List of Gene Therapy Companies with Phase 3 Active Clinical Trial - 73

Our name, 2seventy bio, reflects why we do what we do - TIME. Cancer rips time away, and our goal is to work at the maximum speed of translating human thought into action – 270 miles per hour – to give the people we serve more time. We are building the leading immuno-oncology cell therapy company, focused on discovering and developing new therapies that truly disrupt the cancer treatment landscape With a deep understanding of the human body’s immune response to tumor cells and how to translate cell therapies into practice, we’re applying this knowledge to deliver next generation cellular therapies that focus on a broad range of hematologic malignancies, including the first FDA-approved CAR T cell therapy for multiple myeloma, as well as solid tumors. Our research and development is focused on delivering therapies that are designed with the goal to “think” smarter and faster than the disease. Importantly, we remain focused on accomplishing these goals by staying genuine and authentic to our “why” and keeping our people and culture top of mind every day.

4DMT is a clinical stage biopharma company inventing and developing innovative products to unlock the full potential of genetic medicine to treat large market and rare diseases. Our vector discovery platform Therapeutic Vector Evolution harnesses the power of directed evolution, a Nobel Prize-winning technology, with approximately one billion synthetic AAV capsid-derived sequences to invent customized and evolved vectors with improved therapeutic profiles compared to wild-type vectors. Our product design, development and manufacturing engine empowers us to efficiently create our valuable and diverse product pipeline. This combination of bold innovation and relentless execution gives 4DMT the capability to revolutionize genetic medicines and to strive for potential curative therapies. We have built a deep portfolio of genetic medicine product candidates, with five product candidates in clinical trials for seven patient populations. We are taking genetic medicine to new heights and into new disease areas to overcome challenges not addressed by existing medicines. This incredible challenge drives our relentless and talented team. Our people are the essence of our inspiring, innovative and collaborative environment that encourages individual contributions and provides opportunities for career development. We seek the best in the industry who identify with our mission and our bold and innovative results-driven approach. Our 4 Guiding Principles drive the way we work together: - Dare to Cure - Break Boundaries - Beyond Yourself - Prepare & Execute Relentlessly Join us in creating the future of genetic medicine.

Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Prademagene zamikeracel (pz-cel) is Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets currently in development for recessive dystrophic epidermolysis bullosa. The Company’s fully integrated cell and gene therapy cGMP manufacturing facility served as the manufacturing site for pz-cel used in its Phase 3 VIITAL™ trial, and is capable of supporting commercial production of pz-cel upon FDA approval. The Company’s development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona’s novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases.

ADARx Pharmaceuticals is a newly launched next-wave genetic medicine company focusing on oligonucleotide therapeutics for inhibition, degradation and base editing of mRNA transcripts. With RNA as the target of our drug discovery, we are capable of working on a wide range of diseases and are currently focusing on genetic, cardiometabolic, and central nervous system (CNS) diseases. We are driven by our mission to turn cutting-edge science into life-saving therapeutics and our desire to provide hope for patients with intractable disease.

Adverum Biotechnologies, Inc., a clinical-stage gene therapy company, engages in developing gene therapy product candidates to treat ocular and rare diseases. Its pipeline of product candidates includes ADVM-022, an adeno- associated virus (AAV). 7m8-aflibercept for the treatment of wet age-related macular degeneration; ADVM-043, an investigational gene therapy candidate for the treatment of alpha-1 antitrypsin deficiency; and ADVM-053, a preclinical gene therapy product candidate for the treatment of hereditary angioedema. The company has collaboration agreements with Editas Medicine, Inc. to leverage its proprietary AAV vectors for genome editing technologies to treat up to five inherited retinal diseases; and Regeneron Pharmaceuticals, Inc. for the development of up to eight distinct ocular therapeutic targets. The company was formerly known as Avalanche Biotechnologies, Inc. and changed its name to Adverum Biotechnologies, Inc. in May 2016. Adverum Biotechnologies, Inc. was founded in 2006 and is headquartered in Redwood City, California.

At ALK, we go to work every day to make a difference for the many people with allergies around the world. It is our ambition to make life with allergy surprisingly simple for as many people as possible, and we build upon our strong heritage as a pioneer in allergy immunotherapy, when we continue to innovate, develop and market new products and solutions to help improve the health of people with allergies. Every day, our employees contribute to our aspiration of becoming the world leader in allergy by coming to work to do their best in the pursuit of providing new and better allergy treatments and solutions. We work in a high-energy, international setting where it is imperative that our people thrive and develop. Creativity, influence and opportunities are key elements in developing our people. ALK is a global company with production sites in Denmark, France, Spain and the United States. ALK was founded in Copenhagen in 1923 and today we employ around 2,900 people around the world. Our global headquarters is located in beautiful surroundings in Hørsholm, north of Copenhagen. Read more about ALK at www.alk.net

American Gene Technologies® International Inc. (AGT™) is a private biotechnology company pursuing cures and treatments for infectious diseases, cancers, and monogenic disorders. AGT™'s emerging gene and cell therapeutics programs are designed to: ✅ Create immunotherapy for HIV disease with gene-modified T cells ✅ Activate innate T cell immunity capable of killing all tumor cells with its vector used in immuno-oncology ✅ Treat PKU by bringing a functional gene into cells that express a dysfunctional gene due to mutation ✅ Advance clinically to provide human proof of concept For over a decade, AGT™ has developed, tested, and banked thousands of lentiviral vectors that can be adapted to the specific needs of new target diseases. AGT™ has developed gene therapy for diseases using this rapid gene delivery platform. GENE THERAPIES AGT™’s platform allows it to pursue clinical “cures” for complex diseases. The company has developed patent-protected gene therapeutics that are breakthroughs in medicine. *** See our extensive award winning patents on our website *** HIV/AIDS Immunotherapy for HIV disease with gene-modified T cells IMMUNO-ONCOLOGY Activates innate T cell immunity capable of killing all tumor cells with its vector used in immuno-oncology PHENYLKETONURIA (PKU) Treats PKU by bringing a functional gene into cells that express a dysfunctional gene due to mutation AGT™'s Core Objectives ✔️ Leverage our lentivirus platform to create highly-effective targeted genetic medicines ✔️ Create solutions for patients across a spectrum of infectious diseases, cancers and inherited disorders ✔️ Collaborate with leading medical researchers from around the globe To learn more and get in touch with American Gene Technologies® Inc., contact us today at https://www.americangene.com/contact-us/

Amicus Therapeutics, Inc., a biotechnology company, engages in the discovery, development, and commercialization of therapies to treat a range of rare and orphan diseases. The company offers Galafold, an orally administered small molecule pharmacological chaperone for the treatment of Fabry disease. It is also conducting Phase 3 (ATB200-03) clinical study of AT-GAA for Pompe disease. The company has collaboration and license agreements with Nationwide Children's Hospital and University of Pennsylvania. Amicus Therapeutics, Inc. was founded in 2002 and is headquartered in Philadelphia, Pennsylvania.

Arcellx, Inc., a clinical-stage biotechnology company, engages in the development of various immunotherapies for patients with cancer and other incurable diseases. The company’s lead ddCAR product candidate is CART-ddBCMA, which is in phase 1 clinical trial for the treatment of patients with relapsed or refractory (r/r) multiple myeloma (MM). It is also developing ACLX-001, an immunotherapeutic combination composed of ARC-T cells and bi-valent SparX proteins targeting BCMA to treat r/r MM; ACLX-002 and ACLX-003 for treating r/r acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS); and other AML/MDS product candidates, as well as solid tumor programs. The company was formerly known as Encarta Therapeutics, Inc. and changed its name to Arcellx, Inc. in January 2016. Arcellx, Inc. was incorporated in 2014 and is headquartered in Gaithersburg, Maryland.

Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing.

Beacon Therapeutics is an ophthalmic gene therapy company founded in 2023 to restore and improve the vision of patients with a range of prevalent and rare retinal diseases that result in blindness. The Company has an established scientific foundation that combines a late-stage development candidate to treat X-linked retinitis pigmentosa (XLRP), as well as two preclinical programs, one targeting dry age-related macular degeneration (AMD) and another in-licensed from the University of Oxford targeting cone-rod dystrophy (CRD), an inherited retinal disease. Lead development candidate AGTC-501, is a gene therapy program in Phase II clinical trials for the treatment of XLRP, an inherited monogenic recessive disorder that causes progressive vision loss in boys and young men. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. AGTC-501 expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss. Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space. The Company is supported by funds from Syncona and additional investors including Oxford Science Enterprises.

We are a clinical-stage regenerative medicine company developing novel therapeutics for cardiovascular diseases with large unmet medical needs. BioCardia is developing proprietary comprehensive biotherapeutic solutions for cardiovascular disease in its CardiAMP™ and CardiALLO™ therapies. Clinical results supporting both therapeutic programs are compelling and build upon the most rigorous data in the field to date with intramyocardial delivery of marrow-derived cells. These programs are enabled by the Company's Helix™ transendocardial delivery systems and Morph® vascular access products, which are partnered to enable other promising biotherapeutic programs.

bluebird bio, Inc., a biotechnology company, engages in researching, developing, and commercializing of transformative gene therapies for severe genetic diseases and cancer. Its gene therapy programs include LentiGlobin for the treatment of ß-thalassemia and sickle cell disease; and Lenti-D for the treatment of cerebral adrenoleukodystrophy. The company’s product candidates in oncology include bb2121 and bb21217, which are chimeric antigen receptor T (CAR T) cell product candidates for the treatment of multiple myeloma. It has a strategic collaboration with Bristol-Myers Squibb to discover, develop, and commercialize disease-altering gene therapies in oncology; and Regeneron Pharmaceuticals, Inc. to discover, develop, and commercialize various immune cell therapies for cancer. The company also has collaborations with Medigene AG to discover T cell receptor (TCR) product candidates in the field of cancer; Gritstone Oncology, Inc. to discover TCR product candidates in the field of cancer; TC BioPharm Limited to research and develop gamma delta CAR T cells directed at hematologic and solid tumor targets; Forty Seven, Inc. to pursue clinical proof-of-concept for an antibody-based conditioning regimen; and Novo Nordisk A/S to develop in vivo genome editing treatments for genetic diseases, as well as clinical trial collaboration with Magenta Therapeutics. The company was formerly known as Genetix Pharmaceuticals, Inc., and changed its name to bluebird bio, Inc. in September 2010. bluebird bio, Inc. was founded in 1992 and is headquartered in Cambridge, Massachusetts.

BriaCell Therapeutics Corp. (Nasdaq: BCTX, BCTXW) (TSX: BCT) is a clinical-stage biotechnology company specializing in targeted immunotherapies for cancer. BriaCell achieved positive proof-of-concept in a Phase I/IIa clinical trial for Bria-IMT™ in patients with advanced breast cancer. BriaCell is conducting a Phase I/IIa clinical trial of Bria-IMT™ in combination with a PD-1 inhibitor, retifanlimab (provided by Incyte under a collaboration agreement). Additionally, BriaCell is developing Bria-OTS™, the first “off-the-shelf” personalized immunotherapy for advanced breast cancer.

At Candel Therapeutics, we are developing immunotherapies that induce immunogenic cell death in cancer cells at the site of injection, unmasking tumor neo-antigens within an activated microenvironment. This process leads to a systemic, durable immune response against the tumor, with the potential to change disease outcomes across a variety of indications. Candel’s products are designed to improve survival while maintaining quality of life – from early- to late-stage disease.

Capricor Therapeutics, Inc., a biotechnology company, focuses on the development of transformative cell- and exosome-based therapeutics for the treatment and prevention of a broad spectrum of diseases. The company’s lead candidate, CAP-1002, an allogeneic cardiac-derived cell therapy, which has completed phase II clinical trial for the treatment of patients with late-stage Duchenne muscular dystrophy (DMD); CAP-1002, which is in Phase II clinical trial for the treatment of cytokine storm associated with COVID-19; and completed various trials investigating the use of CAP-1002 for the treatment of cardiac conditions, including heart failure and post myocardial infarction with cardiac dysfunction. It is also developing CAP-2003 that is in pre-clinical development for the treatment of trauma related injuries and conditions; and two vaccine candidates, which are in preclinical stage for the potential prevention of COVID-19. Capricor collaborates with Lonza Houston, Inc. for the development of CAP-1002, its cell therapy candidate for the treatment of DMD and other indications. The company was founded in 2005 and is headquartered in Beverly Hills, California.

Cartesian Therapeutics is a fully integrated, clinical-stage biopharmaceutical company developing novel cell and gene immunotherapies to treat cancer and autoimmune diseases.

Castle Creek Biosciences, Inc., a late-stage cell and gene therapy company, is using two lentiviral platforms to develop and commercialize therapies for genetic diseases.

CellTrans is pioneering cell therapies for clinical application.

CG Oncology, Inc. (Nasdaq: CGON), is a late-stage clinical biopharmaceutical company focused on developing and commercializing a potential backbone bladder-sparing therapeutic for patients afflicted with bladder cancer. We see a world where urologic cancer patients can benefit from our innovative oncolytic immunotherapies to live with dignity and have an enhanced quality of life. Our lead candidate, cretostimogene grenadenorepvec, is an intravesically delivered oncolytic immunotherapy agent in a Phase 3 trial for the treatment of BCG-unresponsive non-muscle invasive bladder cancer. Cretostimogene grenadenorepvec is also in a Phase 2 study in combination with KEYTRUDA® (pembrolizumab) in the same indication. Other types of bladder cancer are being evaluated with cretostimogene grenadenorepvec in combination with OPDIVO® (nivolumab).

Complement Therapeutics GmbH (CTx) is an early-stage biotechnology company focused on the research and development of novel therapeutics for complement-mediated diseases. The Company is a spinout from the University of Manchester and is based on the pioneering research of its founders into novel targets within the complement cascade. Our lead investigational product (CTx001) is being evaluated as a potential gene therapy for dry age-related macular degeneration/geographic atrophy, a leading cause of blindness. Additional programmes will evaluate potential therapeutic opportunities in other complement-mediated conditions. The Company is also developing a unique quantification methodology, the Complement Precision Medicine (CPM) platform, to enable quantification of over 30 complement cascade proteins enabling more precise diagnosis and monitoring of disease.

CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom.

DalCor Pharmaceuticals: DalCor Pharmaceuticals is pioneering precision medicine for patients with cardiovascular disease. By combining genetic and clinical insights, the company’s development program, dalcetrapib, is intended to reduce fatal and non-fatal myocardial infarction (MI) following a recent acute coronary syndrome and deliver superior cardiovascular outcome in a specific genetic subset of patients.

Dendreon is a commercial-stage biopharmaceutical company and pioneer in the development of immunotherapy. Dendreon’s flagship product, PROVENGE (sipuleucel-T), was the first FDA-approved immunotherapy made from a patient’s own immune cells. More than 40,000 men with advanced prostate cancer have been prescribed PROVENGE in the U.S. since 2010. Dendreon also is evaluating the use of PROVENGE in early-stage prostate cancer, with the hope of curing more men of the disease. Dendreon is headquartered in Seal Beach, Calif.

Elpida Therapeutics is a biopharmaceutical company that focuses on developing treatments for rare diseases, offering hope to children and families affected.

We are ESTEVE, a global pharmaceutical company with one defined mission: improving lives. Founded in 1929 and headquartered in Barcelona, we have a strong international footprint, with affiliates across Spain, Portugal, Italy, Germany, France, the UK, and the USA. As a leader in speciality pharmaceuticals, we focus on creating highly specialized treatments that address significant unmet medical needs. Our expertise extends to Contract Manufacturing, where we produce Active Pharmaceutical Ingredients (APIs) through world-class facilities in Spain, Mexico, and China. Driven by our core values—people matter, transparency, and accountability—we’re committed to innovation, quality, and making a meaningful difference in healthcare. Because we care not only about what we do, but also about how we do it. #ESTEVE #AdvancingHealthTogether #Makeitsustainable #Pharmaceuticals #Innovation #SpecialtyPharma #APIs

Evotec is a biotechnology company that is committed to advancing drug discovery and development. Through flexible business models, we collaborate with pharmaceutical companies, biotechs, foundations, and government agencies on a substantial scale. We believe that by working together, we can make a difference in the lives of patients. Our Pipeline Co-Creation model represents our adaptable, multi-modality, fully integrated end-to-end approach to drive collaborations and services across all phases of drug discovery and development – from discovery of novel targets to achieving Proof of Concept in the clinic and into commercial manufacturing. We aspire to contribute data-driven disease understanding and early disease relevance in humans to bring the probability of success up. We select the right modality, which is then propelled forward on our platforms by our passionate people striving for the fastest and most effective ways towards patient impact. This is enabled by convergence of human ingenuity with data and AI. We also offer specific solutions, products, and CRO/CDMO-like services, always in support of the Biopharma R&D innovator. Our more than 4,000 scientists work closely with numerous partners concurrently, delivering fully integrated research and development portfolios or individual projects with the highest quality standards and efficiency, coupled with great science, passion, engagement, and communication.

Fractyl Health is a leader in creating innovative therapies to address the root cause of type 2 diabetes. We were founded based on groundbreaking research and biological insights showing the key role of the intestine in metabolic control. Our therapies are uniquely designed to target and control this controller for the treatment of metabolic disease. Our lead program, Revita DMR, is a first-in-class procedural therapy that is now in late-stage clinical trials for the treatment of type 2 diabetes. Our team is made up of passionate innovators at the intersection of biology and technology, working to deliver better real-world outcomes for people with metabolic disease.

Genexine has robust R&D pipelines in clinical and pre-clinical stages based on long-acting Fc-fusion technology and therapeutic DNA vaccine technology. In the clinical stage, Genexine has GX-H9 (long-acting human growth hormone, hGH-hyFc) co-developed with Handok for both adult and pediatric GHD, currently finished multinational Phase 2 trials and preparing for global Phase 3 trials. GX-188E therapeutic DNA vaccine for HPV-associated diseases is in Phase 2 in Europe and Korea for Cervical Intraepithelial Neoplasia II/III. Combined with Merck’s Keytruda, GX-188E recently is in Phase 1b/2a trial for cervical cancer. HyLeukin-7 (Immuno-oncology agent, IL-7-hyFc) has finished Phase 1 trial in Korea and received IND approval from US FDA. Currently, Genexine is running more than 7 clinical trials globally. In addition to collaboration with Merck, Genexine has strategic partnerships with leading Asian companies such as Shanghai Fosun Pharma, Tasly Pharma, Simcere Pharma, Kalbe Pharma, and Korean big Pharma companies such as Handok, Green Cross and Yuhan.

Meet your business challenges head on with cloud computing services from Google, including data management, hybrid & multi-cloud, and AI & ML.

Gritgen Therapeutics (here in after designated as "Gritgen") was founded in China and is a biotechnology company focused on gene therapy. They have launched a commercial GMP facility in China and have opened a good manufacturing practice (GMP) facility in Suzhou Industrial Park. Gritgen provides an important vector foundation for the company to realize gene therapy for hemophilia A. The company has also launched a Phase 1 clinical trial to assess the safety of gene therapy for hemophilia A.

Hansa Biopharma, is a pioneering commercial-stage biopharmaceutical company on a mission to develop and commercialize innovative and life altering treatments for patients with rare immunological conditions. Hansa scientists are working on developing immunomodulatory treatments for organ transplants, rare IgG-mediated autoimmune conditions, and gene therapy, as well as exploring the potential application of the technology platform in oncology.

Immatics N.V., a clinical-stage biopharmaceutical company, focuses on the discovery and development of T cell receptor (TCR) based immunotherapies for the treatment of cancer in the United States. The company is developing targeted immunotherapies with a focus on treating solid tumors through two distinct therapeutic modalities, such as adoptive cell therapies (ACT) and antibody-like TCR Bispecifics. Its ACTengine product candidates include IMA201 that targets melanoma-associated antigen 4 or 8 in patients with solid tumors; IMA202 that targets melanoma-associated antigen 1 in patients with various solid tumors, including squamous non-small cell lung carcinoma and hepatocellular carcinoma; IMA203 that targets preferentially expressed antigen in melanoma in adult patients with relapsed and/or refractory solid tumors; and IMA204, an anti-tumor therapy that targets the malignant tumor cell. Its TCR Bispecifics product candidates include IMA401, a cancer testis antigen for the treatment of solid tumor; and IMA402 for the treatment of solid and hematological malignancies. The company also develops IMA101, a multi-target precision immunotherapy; and IMA301, an off-the-shelf ACT. It has a strategic collaboration agreement with GlaxoSmithKline plc to develop novel adoptive cell therapies targeting multiple cancer indications; MD Anderson Cancer Center to develop multiple T cell and TCR-based adoptive cellular therapies; Celgene Switzerland LLC to develop novel adoptive cell therapies targeting multiple cancers; Genmab A/S to develop T cell engaging bispecific immunotherapies targeting multiple cancer indications; Amgen Inc.; and MorphoSys to develop novel antibody-based therapies against various cancer antigens that are recognized by T cells. Immatics N.V. is headquartered in Tubingen, Germany.

Immutep is a globally active biotechnology company that is a leader in the development of LAG-3 related immunotherapeutic products for the treatment of cancer and autoimmune disease. Immutep is dedicated to leveraging its technology and expertise to bring innovative treatment options to market for patients and to maximize value to shareholders.

IMUNON is a fully integrated, clinical stage biotechnology company focused on advancing a portfolio of innovative treatments that harness the body’s natural mechanisms to generate safe, effective, and durable responses across a broad array of human diseases. IMUNON has two platform technologies: Our TheraPlas® platform for the development of immunotherapies and other anti-cancer nucleic acid-based therapies and our PLACCINE platform for the development of nucleic acid vaccines for infectious diseases and cancer. The Company’s lead clinical program, GEN-1, is a DNA-based immunotherapy for the localized treatment of advanced ovarian cancer currently in Phase II development. GEN-1 works by instructing the body to produce safe and durable levels of powerful cancer fighting molecules, such as interleukin-12 (IL-12) and interferon gamma (IFN-γ), at the tumor site. In addition, we are conducting pre-clinical proof-of-concept studies on a nucleic acid vaccine candidate targeting SARS-CoV-2 virus in order to validate our PLACCINE platform. IMUNON’s platform technologies are based on the delivery of nucleic acids with novel synthetic delivery systems that are independent of viral vectors or devices. We will continue to leverage these platforms and to advance the technological frontier of plasmid DNA to better serve patients with difficult to treat conditions.

Innate Pharma S.A. is a commercial stage oncology-focused biotech company dedicated to improving treatment and clinical outcomes for patients through therapeutic antibodies that harness the immune system to fight cancer. Innate Pharma’s commercial-stage product, Lumoxiti, in-licensed from AstraZeneca, was approved by the FDA in September 2018. Lumoxiti is a first-in class specialty oncology product for hairy cell leukemia (HCL). Innate Pharma’s broad pipeline of antibodies includes several potentially first-in-class clinical and preclinical candidates in cancers with high unmet medical need. Pioneers in the biology of Natural Killer cell, Innate Pharma has expanded its expertise in the tumor microenvironment and tumor-antigens, as well as antibody engineering. This innovative approach has resulted in a diversified proprietary portfolio and major alliances with leaders in the biopharmaceutical industry including Bristol-Myers Squibb, Novo Nordisk A/S, Sanofi, and a multi-products collaboration with AstraZeneca.

We are a pioneering, clinical-stage biopharmaceutical company dedicated to developing innovative therapeutics for rare diseases that affect bone health and blood vessel function. Our lead candidate, INZ-701, is an ENPP1 Fc fusion protein enzyme replacement therapy (ERT) designed to increase PPi and adenosine, enabling the potential treatment of multiple diseases caused by deficiencies in these metabolites. By targeting the PPi-Adenosine Pathway, INZ-701 aims to correct pathological mineralization and intimal proliferation, addressing the significant morbidity and mortality in these devastating diseases. It is currently in clinical development for the treatment of ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis.

Insmed Incorporated, a biopharmaceutical company, focuses on the development and commercialization of therapies for patients with serious and rare diseases. The company offers ARIKAYCE for the treatment of Mycobacterium avium complex lung disease as part of a combination antibacterial drug regimen for adult patients in the United States. It also develops INS1007, an oral reversible inhibitor of dipeptidyl peptidase 1 for the treatment of non-cystic fibrosis bronchiectasis and other inflammatory diseases; and INS1009, an inhaled formulation of a treprostinil prodrug for the treatment of rare pulmonary disorders, including pulmonary arterial hypertension. The company was founded in 1988 and is headquartered in Bridgewater, New Jersey.

The Institut Pasteur, a non-profit foundation with recognized charitable status set up by Louis Pasteur in 1887, is today an internationally renowned center for biomedical research with a network of 33 members worldwide. In the pursuit of its mission to tackle diseases in France and throughout the world, the Institut Pasteur operates in four main areas: research, public health, training, and development of research applications. The Institut Pasteur is a globally recognized leader in infectious diseases, microbiology, and immunology, with research focusing on the biology of living systems. Among its areas of investigation are emerging infectious diseases, antimicrobial resistance, certain cancers and brain connectivity diseases. The Institut Pasteur's outstanding research is facilitated by the development of a technological environment of the highest standard, with core facilities for nanoimaging, computational biology and artificial intelligence. Since its inception, 10 Institut Pasteur scientists have been awarded the Nobel Prize for Medicine, including two in 2008 for the 1983 discovery of the human immunodeficiency virus (HIV) that causes AIDS. www.pasteur.fr/en

Intellia Therapeutics is a leading genome editing company whose mission is to develop potentially curative gene editing treatments that can positively transform the lives of people living with severe and life-threatening diseases. We are focused on the development of proprietary therapeutics using a recently developed biological tool known as the CRISPR/Cas9 system. The promise of the CRISPR/Cas9 system is the driving force behind the creation of Intellia. Our founders have a shared belief that the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course. This technology offers the potential for us to develop curative therapeutic options for patients with chronic diseases by addressing the underlying cause of the disease. It can bring new hope and cures to people who now have nowhere to turn for help. Our combination of deep scientific expertise and clinical development experience, along with our leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Intellia has the right people, assets and ambitious vision needed to take full advantage of the CRISPR/Cas9 technology’s attributes – high potency, specificity, simplicity of use, broad applicability, and multifunctional programmability – and accelerate the advancement of curative products into the clinic. View our social media community guidelines at https://www.intelliatx.com/terms-and-conditions/ .

Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an injection for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults; and WAYLIVRA, a treatment for familial chylomicronemia syndrome and familial partial lipodystrophy. It is involved in developing neurology products that include Tominersen for Huntington’s diseases; Tofersen for amyotrophic lateral sclerosis (ALS); IONIS-MAPTRx for Alzheimer's diseases; IONIS-C9Rx for genetic form of ALS; ION859 for Parkinson’s disease; ION464 for multiple system atrophy and Parkinson’s disease; ION541 for amyotrophic lateral sclerosis; and IONIS-DNM2-2.5Rx for centronuclear myopathy. The company is also developing products for cardiometabolic disease, such as IONIS-TTR-LRx for transthyretin amyloidosis and IONIS-GHR-LRx for acromegaly; IONIS-APOCIII-LRx for familial chylomicronemia syndrome and TG- driven diseases; IONIS-APOCIII-LRx for treatment resistant hypertension; Pelacarsen for Lp(a)cardiovascular disease risk reduction; Vupanorsen for sHTG/CVDRR; IONIS-FXI-LRx for clotting disorders; ION449 for cardiovascular diseases; and IONIS-GCGRRx for diabetics. In addition, it develops IONIS-ENAC-2.5Rx for cystic fibrosis and chronic obstructive pulmonary disease; IONIS-PKK-LRx for hereditary angioedema; Danvatirsen for cancer; IONIS-TMPRSS6-LRx for beta-thalassemia and iron toxicity; IONIS-HBVRx for hepatitis B virus; IONIS-AR-2.5Rx for prostate cancer; IONIS-FB-LRx for age-related macular degeneration and IgA nephropathy; and ION357 for retinitis pigmentosa. It has a collaboration agreement with AstraZeneca; GlaxoSmithKline plc; Janssen Biotech, Inc.; Bayer AG; Novartis AG; Pfizer, Inc.; Biogen Inc.; and Roche. Ionis Pharmaceuticals, Inc. was founded in 1989 and is based in Carlsbad, California.

Iovance Biotherapeutics (NASDAQ: IOVA) aims to be the global leader in innovating, developing and delivering tumor infiltrating lymphocyte (TIL) therapies for patients with cancer. We are pioneering a transformational approach to cure cancer by harnessing the human immune system’s ability to recognize and destroy diverse cancer cells in each patient. The Iovance TIL platform has demonstrated promising clinical data across multiple solid tumors. We are committed to continuous innovation in cell therapy, including gene-edited cell therapy, that may extend and improve life for patients with cancer. We are adding to our talented team as we execute our commercial launch, advance our research and development efforts, and build our internal manufacturing capabilities. Iovance is headquartered in the San Francisco Bay Area. Our Iovance Cell Therapy Center (iCTC), a state-of-the-art manufacturing facility, is located in Philadelphia. We also have a research site in Tampa, Florida. Any information expressed by Iovance is subject to the risk factors and information on forward-looking statements contained in its filings with the Securities and Exchange Commission and available here: https://ir.iovance.com/sec-filings

At Kite, a Gilead Company, our singular focus is cell therapy to treat and potentially cure cancer. Our goal is to bring the promise of cell therapy to as many eligible patients as possible who may benefit. Since 2009, Kite has been at the forefront of cancer immunotherapy. Today, we are proud to be the leader in engineered T cell therapy for hematologic malignancies, but we recognize that this is just the beginning. Patients are at the heart of our focus on the cure, which is why we are committed to pursuing cell therapy as a transformative treatment option for as many people with cancer as possible who may benefit. Cancer is personal to us. Unlike traditional pharmaceutical manufacturing, our therapies are not created on an assembly line. Cell therapy specialists at our manufacturing facilities reengineer a patient’s own immune cells to attack and fight their cancer cells — one patient at a time. That sense of connection to the people we serve motivates us daily at Kite. Our corporate headquarters are located in Santa Monica, California, and we have dedicated cell therapy manufacturing facilities in California, Maryland and the Netherlands. If you’re passionate about changing the future of cancer treatment, let’s talk.

At Kolon TissueGene, we are developing advanced cell therapies to target various orthopedic diseases and degenerative disorders.

Krystal Biotech, Inc. is using gene therapy to develop effective and novel treatments for skin diseases. Our goal is to make a meaningful difference in the lives of underserved patient populations with debilitating skin diseases. We work to accomplish that through scientific innovation, operational excellence and believe that “nature operates in the shortest way possible”. (Aristotle)

MED Institute accelerate product development. MED Institute is dedicated to bringing new medical device products to market that are safe and effective for patients. From concept to commercialization, our team has the knowledge and experience to guide your products through the complex steps required for market approval.

OUR MISSION: Deliver Innovation. Empower Discovery. Improve Life. For over 25 years, Mirus Bio has pioneered the development of transfection reagents and proprietary technologies for nucleic acid delivery applications. Our work is both technical and cutting-edge, and we promote a culture of creativity, responsibility, and respect. Our team members are engaged and motivated by the desire to support our customers and partners to achieve strong results. Mirus, together with Gamma Biosciences, is establishing the future of science and medicine by developing products that enable product developers within the cell and gene therapy space. As we approach a new era of addressing and conquering life-altering diseases, we will continue to expand our expertise and leadership in transfection to be integral in the future of advanced therapies.

Nanoscope is focused on proprietary ambient light activated optogenetic therapy to restore vision in people suffering from all forms of Retinitis Pigmentosa.

Neurogene is focused on developing life-changing genetic medicines for patients & families affected by rare, devastating neurological diseases.

Ocugen, Inc. is a biopharmaceutical company focused on discovering, developing, and commercializing gene therapies to cure blindness diseases and developing a vaccine to save lives from COVID-19. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with one drug – “one to many” and our novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema, and diabetic retinopathy. We are co-developing Bharat Biotech’s COVAXIN™ vaccine candidate for COVID-19 in the U.S. market.

Ocuphire Pharma Inc is a clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing innovative small molecule therapies for eye disorders. The company aims to address unmet medical needs in the ocular space, particularly in retinal and refractive disorders. Ocuphire Pharma offers research and development services to create novel treatments for various eye conditions. The company collaborates with pharmaceutical partners to advance its drug candidates through clinical trials and commercialization. Its product pipeline includes Nyxol, an ophthalmic solution for dim light and night vision disturbances; APX3330, an oral inhibitor for diabetic retinopathy and macular edema; APX2009, aimed at treating wet age-related macular degeneration; and Phentolamine Ophthalmic Solution (RYZUMVI), approved for pharmacologically induced mydriasis and under development for other conditions.

Orchard Therapeutics, a Kyowa Kirin company, is a global gene therapy leader focused on ending the devastation caused by genetic and other severe diseases by discovering, developing, and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patient’s own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease with a single treatment. Founded in 2015, Orchard’s roots go back to some of the first research and clinical developments involving HSC gene therapy. Our team has played a central role in the evolution of this technology from a promising scientific idea to a potentially life-transforming reality. Today, Orchard is advancing a pipeline of HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist. For more information, please visit www.orchard-tx.com.

Organogenesis™ is a leading regenerative medicine company focused on the development, manufacture, and commercialization of solutions for the advanced wound care and surgical and sports medicine markets. Organogenesis offers a comprehensive portfolio of innovative regenerative products to address patient needs across the continuum of care.

OSE Immunotherapeutics is an integrated biotech company focused on developing first-in-class assets targeting cancer and inflammatory diseases. A current well-balanced first-in-class clinical pipeline including: • Tedopi® (T-cell specific immunotherapy): Company’s most advanced product; positive results for Ph 3 in NSCLC in secondary resistance after CKI failure. Other sponsored combo Ph 2 trials in solid tumors ongoing. • OSE-279 (anti-PD1) advanced preclinical stage • OSE-127 (humanized mAb antagonist of IL-7R); ongoing Ph 2 in Ulcerative Colitis; ongoing preclinical research in leukemia • VEL-101/FR104 (anti-CD28 mAb) developed in partnership with Veloxis in transplantation; ongoing Ph 1/2 in renal transplant (sponsor Nantes University Hospital); Ph 1 ongoing in the US (sponsor Veloxis). • BI 765063 (anti-SIRPα mAb on CD47/SIRPα pathway) developed in partnership with Boehringer Ingelheim in advanced solid tumors; positive Ph 1 dose escalation results in mono and in combo, in particular with anti-PD-1 antibody ezabenlimab; BI sponsored international Ph 1b clinical trial ongoing in combo with ezabenlimab alone or with other drugs in recurrent/metastatic HNSCC and HCC. OSE Immunotherapeutics expects to generate further significant value from its two proprietary drug discovery platforms, which are central to its ambitious goal to deliver next-generation first-in-class immunotherapeutics: • BiCKI® platform focused on immuno-oncology (IO) is a bispecific fusion protein platform built on the key backbone component of anti-PD1 combined with a new immunotherapy target to increase anti-tumor efficacy. Most advanced BiCKI® candidate is targeting anti-PD1xIL-7. • Myeloid platform focused on optimizing the therapeutic potential of myeloid cells in IO & immuno-inflammation (I&I). OSE-230 (ChemR23 agonist mAb), most advanced candidate generated by the platform, with the potential to resolve chronic inflammation.

Pacira BioSciences Inc. is a leading provider of non-opioid pain management and regenerative health solutions. We are dedicated to offering patients—and the clinicians who treat them—cutting-edge therapies to treat pain with the potential to reduce or eliminate the need for opioids. Founded in 2006 and headquartered in Tampa, FL, Pacira has offices in Parsippany, NJ as well as San Diego, CA. With more than 700 employees who share a passion for delivering improved patient care and reducing the need for opioids, Pacira is here to challenge and transform the way we think about – and treat – pain. We are committed to providing solutions that address unmet medical need and improve clinical results, and empowering patients and healthcare providers to choose non-opioid options. Our non-opioid product portfolio is aimed at opioid minimization, long-lasting pain control, and enhanced recovery. Find out why we aim for change at Pacira.com/commitments

Phoenix Nest Biotech is a biotechnology company that focuses on treating Sanfilippo Syndrome by collaborating with leading academics and utilizing licensed key technologies.

Pierre Fabre Group held by the Pierre Fabre Foundation, is a worldwide company with a unique positioning : the alliance of pharmaceutical and dermocosmetics expertise. This makes Pierre Fabre Group : 💊 A pharmaceutical group with a strong positioning : medical and natural 🥈 The second largest dermo-cosmetics laboratory in the world 🥈 The second largest private French pharmaceutical group 🥇 The market leader in France for products sold over the counter in pharmacies. Our portfolio includes several medical franchises and international brands including ; Pharmaceutical Care Pierre Fabre Oncologie Pierre Fabre Dermatologie Naturactive Eau Thermale Avène Klorane Ducray René Furterer A-Derma Pierre Fabre Oral Care Glytone (US) Darrow (Brasil) Established in the Occitanie region since its creation, we manufacture over 95% of our products in France. We keep innovating passionately with our teams in 2 innovation centers in Brasil and Japan as well as in 6 R&D centers in France. Thanks to our 9,600 employees in 44 subsidiaries and our distribution activities in 120 countries, our group generated €2.7 billion in revenues in 2022, 69% of which is from international business. Pierre Fabre Group has a unique company structure. 86% of the Pierre Fabre Group is held by the Pierre Fabre Foundation, a government-recognized public-interest foundation, while a smaller share is owned by its employees via an employee stock ownership plan. In 2020, the independent organization ECOCERT Environment awarded Pierre Fabre's CSR policy at "excellence" level : - Excellence being the highest maturity level of the ECOCERT 26000 standard.

Precigen (Nasdaq: PGEN) is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cell therapies using precision technology to target the most urgent and intractable diseases in immuno-oncology, autoimmune disorders, and infectious diseases. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well-differentiated unique therapies toward clinical proof-of-concept and commercialization.

ProKidney Corp., a clinical-stage biotechnology, engages in developing cellular therapy candidates. It is developing Renal Autologous Cell Therapy, an autologous homologous cell admixture that is in a Phase III development program, as well as Phase II clinical trials for the treatment of moderate to severe diabetic kidney disease; and Phase I clinical trial for patients with congenital anomalies of the kidney and urinary tract. The company was founded in 2015 and is headquartered in Winston-Salem, North Carolina.

We are ProQR, a biotechnology company dedicated to changing lives by developing RNA therapies for rare genetic diseases. We focus our drug development mainly on a group of blinding disorders affecting the retina, called inherited retinal diseases.

PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines for the treatment of rare disorders. The company offers Translarna (ataluren) and Emflaza (deflazacort) for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients. It is developing Translarna, which is in Phase 2 clinical study for the treatment of nonsense mutation aniridia and nonsense mutation Dravet syndrome/CDKL5; and RG7916 and RO7034067 for the treatment of patients with spinal muscular atrophy, as well as PTC596 and PTC299, a small molecule dihydrooratate dehydrogenase (DHODH) inhibitor that inhibits de novo pyrimidine nucleotide synthesis, which is in Phase 1 clinical development stage to treat cancer patients. The company is also developing gene therapy product candidate that include PTC-AADC for the treatment of Aromatic L-amino acid decarboxylase deficiency. It has collaborations with F. Hoffman-La Roche Ltd and Hoffman-La Roche Inc., and the Spinal Muscular Atrophy Foundation to develop and commercialize compounds identified under its spinal muscular atrophy sponsored research program; research collaboration with Massachusetts General Hospital for the treatment of rare genetic disorders resulting from pre-mRNA; and Akcea Therapeutics, Inc. to commercialize Tegsedi and Waylivra for the treatment of rare diseases in Latin America and the Caribbean. PTC Therapeutics, Inc. was founded in 1998 and is headquartered in South Plainfield, New Jersey.

Replimune Group, Inc., a biotechnology company, develops oncolytic immune-gene therapies to treat cancer. It uses its proprietary Immulytic platform to design and develop product candidates that are intended to activate the immune system against cancer. The company's lead product candidate is RP1, a selectively replicating version of herpes simplex virus 1, which is in Phase I/II clinical trials for a range of solid tumors; and that is in Phase II clinical trials for patients with cutaneous squamous cell carcinoma. It is also developing RP2, which is in Phase I clinical trials for an anti-CTLA-4 antibody-like protein in order to block the inhibition of the immune response otherwise caused by CTLA-4; and RP3 to express immune-activating proteins that stimulate T cells. Replimune Group, Inc. was founded in 2015 and is headquartered in Woburn, Massachusetts.

Rhythm is a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity. The company recently announced topline results from pivotal Phase 3 clinical trials in POMC deficiency obesity and LEPR deficiency obesity. Rhythm is also conducting a pivotal Phase 3 trial in Bardet-Biedl syndrome and Alström syndrome. The company is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study in additional rare genetic disorders of obesity, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of these disorders. For healthcare professionals, visit www.UNcommonObesity.com for more information.

Sarepta Therapeutics, headquartered in Cambridge, Massachusetts, is a global biotechnology company on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For information on our Community Guidelines, please visit sarepta.com/community-guidelines. We want to share a reminder with all job seekers and candidates regarding the persistence of recruiting fraud. Please read a message about recruiting fraud and steps you can take to protect yourself here: https://www.sarepta.com/recruiting-fraud

Seagen Inc., a biotechnology company, develops and commercializes therapies for the treatment of cancer in the United States and internationally. The company offers ADCETRIS, an antibody-drug conjugate (ADC) for the treatment of patients with Hodgkin lymphoma or CD30-positive T-cell lymphomas; PADCEV, an ADC targeting Nectin-4 for the treatment of advanced or metastatic urothelial cancer; and TUKYSA, an oral small molecule tyrosine kinase inhibitor for the treatment of adult patients with advanced unresectable or metastatic HER2-positive breast cancer. It also develops TIVDAK for metastatic cervical cancer and other solid tumors; Ladiratuzumab Vedotin, an ADC targeting LIV-1 for metastatic breast cancer and solid tumors; Disitamab Vedotin, a novel HER2-targeted ADC; and SEA-CD40, SEA-TGT, SEA-BCMA, and SEA-CD70 for various cancer diseases. Seagen Inc. has collaboration agreements with Takeda Pharmaceutical Company Limited; Agensys, Inc.; Genmab A/S; Merck; and RemeGen, Co. Ltd. The company was formerly known as Seattle Genetics, Inc. and changed its name to Seagen Inc. in October 2020. Seagen Inc. was incorporated in 1997 and is headquartered in Bothell, Washington. On Dec. 14, 2023, Pfizer completed the acquisition of Seagen.

Sequirus is one the largest cell-based influenza vaccine producer in the world. The company also makes antivenoms, and Q fever vaccine.

Synbio Technologies aims to become the most trusted provider of DNA solutions that empower scientific discoveries by providing highly-accurate and cost-effective synthetic biology services and products for researchers around the world. We have developed a full range of DNA reading (sequencing), DNA writing (synthesis), and DNA editing (engineering) capabilities for various applications, including diagnostic DNA probes, precision medicine, protein production, antibody discovery, vaccine development, novel enzymes, molecular breeding, biofuel implication, and more. Our scientific capabilities cover all facets of DNA synthesis and engineering, offering a full range of support for molecular biology work, including but not limited to, oligo synthesis, gene synthesis, subcloning & PCR cloning, plasmid preparation, mutagenesis, DNA variant libraries, CRISPR sgRNA, protein expression and purification in multiple systems. Relying on our strong DNA sequencing, synthesis, engineering technology platform, and expertise in bioinformatics analysis, we have developed proprietary algorithms to facilitate and expedite the antibody discovery process. Our antibody discovery services include hybridoma sequencing, immune repertoire sequencing, antibody design and production, AI-guided heavy chain and light chain pairing, antibody humanization, affinity maturation, and sdAb.

Thermo Fisher Scientific Inc. is the world leader in serving science, with annual revenue of approximately $40 billion. Our Mission is to enable our customers to make the world healthier, cleaner and safer. Whether our customers are accelerating life sciences research, solving complex analytical challenges, increasing productivity in their laboratories, improving patient health through diagnostics or the development and manufacture of life-changing therapies, we are here to support them. Our global team delivers an unrivaled combination of innovative technologies, purchasing convenience and pharmaceutical services through our industry-leading brands, including Thermo Scientific, Applied Biosystems, Invitrogen, Fisher Scientific, Unity Lab Services, Patheon and PPD.

Leading with purpose. Every day. “Ultragenyx was founded to advance innovative medicines for rare and ultrarare diseases that have never been treated before. We are delivering transformative therapies across multiple indications, and we have one of the most robust and diverse clinical pipelines in rare disease. Our focus is on doing the right things for patients both during development and commercialization to deliver on the promise of these therapies in a way that's meaningful for rare disease communities.” Emil D. Kakkis, M.D., Ph.D. Chief Executive Officer and President We have an inclusive culture of value and respect. Ultragenyx brings novel products to those living with rare and ultrarare diseases with a focus on debilitating genetic diseases. Founded in 2010, we have rapidly built a diverse portfolio of approved therapies and products aimed at diseases with a high unmet medical need for which many have no approved therapies. It takes courage, care, talent, and dedication to make a meaningful impact for those living with rare diseases, their families and the rare disease community. We put our people first, so they can take care of the rare disease community. We purposefully cultivate and nurture a dynamic, supportive work environment where we encourage every team member to share their new ideas and help us unlock more possibilities. Our team is made up of exceptional and diverse people with a strong passion and commitment to helping those living with rare diseases. By embracing generosity, curiosity, inclusion, and humility we are constantly learning together—fostering an environment that supports profound growth and fulfillment. If you want to have a meaningful impact, do the best work of your career while having fun and growing professionally and personally, come join our team! Visit our community guidelines at: https://ultragenyx.co/community

This page is no longer active or monitored. We are now Sumitomo Pharma America (SMPA), a science-based, technology-driven biopharmaceutical company focused on delivering therapeutic and scientific breakthroughs in areas of critical patient need. See pinned post for our new page and follow us!⠀⠀⠀ ⠀⠀⠀⠀ ⠀⠀⠀⠀ ⠀⠀⠀⠀ ⠀⠀⠀⠀ ⠀⠀⠀⠀ ⠀⠀⠀⠀ ⠀ ==== We aim to advance urologic patient care through bold innovation, based on diversity of thought, openness to new ideas, adaptation to change, integrity and compassion. We develop innovative therapies and participate in exceptional licensing opportunities. Our goal is to be the leading specialty company: recognized and trusted by patients and providers for our commitment to urology. Urologic conditions can have a major impact on daily life, with those affected often suffering in silence as their conditions are overlooked, misunderstood or not adequately treated. Read more about our community guidelines: https://urovant.com/community-guidelines/

Vericel is a leading provider of advanced therapies for the sports medicine and severe burn care markets. The Company combines innovations in biology with medical technologies, resulting in a highly differentiated portfolio of innovative cell therapies and specialty biologics that repair injuries and restore lives. We currently market two autologous cell therapy products and one specialty biologic product in the United States. Personal integrity, teamwork, collaboration, and innovative technology are the foundations of our work. We seek to practice transparency in our clinical trials and research, and in our relationships with each other, our patients, and the investors who support us. PRIVACY AND COMMUNITY GUIDELINES When you engage with Vericel social media channels and our content providers on social media, you also agree to abide by the following Community Guidelines (found here: https://bit.ly/3pg3UKQ), which apply in addition to the Terms and Conditions or other legal notices specific to each social media channel. Please note that third-party social media channels also have access to information you share via social media. For more details, read LinkedIn's Privacy Policy here: https://bit.ly/3GF2r5Y. For additional information about privacy at Vericel, please see our Privacy Policy (bit.ly/43ErZcx) and Terms and Conditions of Use (bit.ly/4568Cdx). If you engage with us, you consent and give Vericel permission to communicate with you and to use any information, text and media associated with the interaction or post.