List of Gene Editing/CRISPR Companies with Phase 2 Active Clinical Trial - 50

Affini-T is a leading precision immunotherapy company targeting core oncogenic driver mutations to develop potentially curative therapies for patients with solid tumors. Our differentiated cell therapy platforms harness state-of-the-art engineering, synthetic biology, and gene editing capabilities to target even the most devastating cancer-driving mutations, beginning with KRAS. We leverage these tools to optimize T cell functions and rewrite the rules of the solid tumor microenvironment, enabling the potential for sustained clinical outcomes in patients. Building on the world-class innovation inherent in our leadership team, founders and technologies, we are powered to develop transformational medicines that last.

Alaunos Therapeutics (previously Ziopharm Oncology, Inc.) a clinical-stage biopharmaceutical company, focuses on discovering, acquiring, developing, and commercializing a portfolio of immuno-oncology therapies to treat patients with cancer. The company develops Sleeping Beauty platform, which is based on the non-viral genetic engineering of immune cells using a transposon/transposase system to engineer T-cells outside of the body for infusion; and Controlled IL-12 to stimulate expression of interleukin 12 or IL-12, a master regular of the immune system, in a controlled manner to focus the patient’s immune system to attack cancer cells. Its product candidates include T cell receptor + T therapies to target solid tumors; chimeric antigen receptor + T cell therapies targeting CD19 for hematologic malignancies; and Ad-RTS-hIL-12 plus veledimex, a gene delivery system to regulate production of IL-12 to treat patients with recurrent glioblastoma multiforme in adults. The company has a license agreement with PGEN Therapeutics, Inc.; research and development agreement with The University of Texas MD Anderson Cancer Center; a patent license agreement with the National Cancer Institute; and a cooperative research and development agreement with the National Cancer Institute. The company was formerly known as ZIOPHARM Oncology, Inc. and changed its name to Alaunos Therapeutics, Inc. in January 2022. Alaunos Therapeutics, Inc. is based in Houston, Texas.

Allogene Therapeutics, Inc., a clinical stage immuno-oncology company, develops and commercializes genetically engineered allogeneic T cell therapies for the treatment of cancer. The company is developing UCART19, an allogeneic chimeric antigen receptor (CAR) T cell product candidate, which is in Phase I clinical trials for the treatment of pediatric and adult patients with R/R CD19 positive B-cell ALL; ALLO-501, an anti-CD19 allogeneic CAR T cell product candidate that is in Phase I clinical trial for the treatment of R/R non-Hodgkin lymphoma; and ALLO-501A for the treatment R/R large B-cell lymphoma or transformed follicular lymphoma. It is also developing ALLO-715, an allogeneic CAR T cell product candidate that is in a Phase 1 clinical trial for treating R/R multiple myeloma; ALLO-819, an allogeneic CAR T cell product candidates for the treatment of acute myeloid leukemia; ALLO-647, an anti-CD52 monoclonal antibody; CD70 to treat renal cell cancer; and DLL3 for the treatment of small cell lung cancer and other aggressive neuroendocrine tumors. The company has license and collaboration agreements with Pfizer Inc.; Servier; Cellectis S.A.; and Notch Therapeutics Inc., as well as clinical trial collaboration agreement with SpringWorks Therapeutics, Inc. Allogene Therapeutics, Inc. also has a strategic collaboration agreement with The University of Texas MD Anderson Cancer Center for the preclinical and clinical investigation of AlloCAR T candidates across a portfolio of hematologic and solid tumors. The company was founded in 2017 and is headquartered in South San Francisco, California.

Arbor Bio is a next-generation gene editing company focused on discovering and developing potentially curative genomic medicines, with the most extensive toolbox of proprietary genomic editors in the industry. We have the unique ability to work backward from disease pathology and choose the optimal editors or combination of editors for the disease in question.

ArsenalBiosciences, Inc., is a privately held, clinical stage programmable cell therapy company focused on the realization of solid tumor cell therapy to defeat cancer. Our discovery engine comprises precise CRISPR-enabled genome editing, integrated circuits incorporating logic gates for improved tumor targeting, and therapeutic enhancements enabling multiple pharmaceutical functions. With our programmable and computationally driven approach, we aim for enhanced and broader efficacy, increased patient safety, reduced provider costs and expanded market access. Our team is engineering living medicines to attack cancer’s inherent multi-faceted nature and bring dramatic improvements to the lives of patients.

Ascidian Therapeutics is redefining the treatment of disease by rewriting RNA. By editing exons at the RNA level, Ascidian therapies enable precise post-transcriptional editing of genes, resulting in full-length, functional proteins at the right levels, in the right cells, at the right time. With active discovery and preclinical programs in ophthalmology, neurological and neuromuscular disorders, and other rare diseases, Ascidian is opening new therapeutic possibilities for patients in need of breakthroughs.

ASC Therapeutics focuses on the development of curative therapies for rare diseases. Our gene, CRISPR and cell therapies were developed at Applied StemCell, a life sciences company for over 12 years, including proprietary gene editing technologies. Our development pipeline includes several preclinical stage projects focusing on monogenic blood disorders that exhibit a high genetic penetrance and conditions requiring immunomodulatory interventions. Contact us for partnership opportunities

Atara Biotherapeutics Inc., is a leading allogeneic T-cell immunotherapy company pioneering the development of transformative off-the-shelf therapies for patients with serious diseases including solid tumors, hematologic cancers, and autoimmune diseases.  With our lead program receiving marketing authorization in Europe, Atara is the most advanced allogeneic T-cell immunotherapy company and intends to rapidly deliver off-the-shelf treatments to patients with high unmet medical need. Our platform leverages the unique biology of EBV T cells and has the capability to treat a wide range of EBV-associated diseases, or other serious diseases through incorporation of engineered CARs (chimeric antigen receptors) or TCRs (T-cell receptors). Atara is applying this one platform, which does not require TCR or HLA gene editing, to create a robust pipeline including: tab-cel for Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) and other EBV-driven diseases; ATA188, a T-cell immunotherapy targeting EBV antigens as a potential treatment for multiple sclerosis; and multiple next-generation chimeric antigen receptor T-cell (CAR-T) immunotherapies for both solid tumors and hematologic malignancies.

Beam Therapeutics Inc., a biotechnology company, engages in developing precision genetic medicines for patients suffering from serious diseases in the United States. The company is developing therapies for the development of sickle cell disease and beta-thalassemia; CAR-T cell therapies for pediatric T-cell acute lymphoblastic leukemia and pediatric acute myeloid leukemia; therapies for alpha-1 antitrypsin deficiency and glycogen storage disorder 1A; and therapies for ocular and central nervous system disorders. It has a research and clinical collaboration agreement with Magenta Therapeutics, Inc. Beam Therapeutics Inc. was founded in 2017 and is based in Cambridge, Massachusetts.

Be Bio is pioneering Engineered B Cell Medicines (BCMs) to dramatically improve the lives of patients who are living with Hemophilia B and other genetic diseases, cancer, and other serious conditions. With eyes locked on the patient, our team of purpose-driven scientists, technologists, manufacturing experts and business builders collaborate to create a bold new class of cell therapies.

Capsida Biotherapeutics Inc. is an industry-leading gene therapy platform company creating a new class of targeted, non-invasive gene therapies for patients with debilitating and life-threatening genetic disorders. Capsida's technology allows for the targeted penetration of cells and organs, while limiting collateral impact on non-targeted cells and organs, especially the liver. This technology allows for the delivery of the gene therapy in a non-invasive way through intravenous administration. Capsida's technology is protected by a growing intellectual property portfolio which includes more than 30 patent applications and one issued U.S. patent 11,149,256. The company is exploring using the technology across a broad range of life-threatening genetic disorders. Its initial pipeline consists of multiple neurologic disease programs. The company has strategic collaborations with AbbVie and CRISPR, which provide independent validation of Capsida's technology and capabilities. Capsida is a multi-functional and fully integrated biotechnology company with proprietary adeno-associated virus (AAV) engineering, multi-modality cargo development and optimization, translational biology, process development and state-of-the-art manufacturing, and broad clinical development experience. Capsida's biologically driven, high-throughput AAV engineering and cargo optimization platform originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at the California Institute of Technology.

CARsgen is a biopharmaceutical company with operations in China and the U.S. focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors. The company has built an integrated cell therapy platform with in-house capabilities that span target discovery, antibody development, clinical trials, and commercial-scale manufacturing. CARsgen has internally developed novel technologies and a product pipeline with global rights to address major challenges of CAR T-cell therapies, such as improving the safety profile, enhancing the efficacy in treating solid tumors, and reducing treatment costs. Our vision is to become a global biopharmaceutical leader that brings innovative and differentiated cell therapies to cancer patients worldwide and makes cancer curable.

Cellectis is a global clinical-stage biopharmaceutical company. Pioneers and innovators in our field, our mission is to develop innovative treatments for patients with unmet medical needs. With 25 years of expertise, we have the best-in-class gene editing platform focusing on immuno-oncology, and gene therapy. Through our efficient and precise TALEN® technology, we create allogeneic CAR-T cells capable of recognizing and combating cancer cells. Today, our three clinical programs target patients with B-cell acute lymphoblastic leukemia (B-ALL), non-Hodgkin lymphoma (NHL) and acute myeloid leukemia (AML). We are fully integrated and a leader in end-to-end gene editing, allogeneic CAR T-cell companies. With our in-house manufacturing, we control our gene and cell therapy process from start to finish with starting materials produced in Paris (France) and CAR-T therapy products created in Raleigh, NC (USA). We also have several ongoing strong collaborations, based on our TALEN® technology, with leading cell & gene therapy companies, including our recent partnership with AstraZeneca, to develop new product candidates in oncology, immunology, and treatment of rare diseases. At Cellectis, we are committed to a cure. Cellectis headquarters are in Paris, France, with additional locations in New York, New York and Raleigh, North Carolina. Cellectis is listed on the Nasdaq Global Market (ticker: CLLS) and on Euronext Growth (ticker: ALCLS). To find out more about us, visit our website: www.cellectis.com. To learn more about our community guidelines, visit: http://www.cellectis.com/en/social-media-guidelines/ Follow our other social media accounts: @cellectis on LinkedIn on X (formerly Twitter) . TALEN® is a registered trademark owned by the Cellectis Group.

Chordia Therapeutics is a clinical-stage biotech company engaged in the research and development of novel therapies for cancers with high unmet medical needs. The Company was spun out from Takeda Pharmaceutical in November 2017.

EdiGene is a clinical-stage company focused on translating gene-editing technologies into transformative genetic medicines for patients with significant unmet medical needs. Anchored by its proprietary technologies of gene editing, bioinformatics, and high throughput genome-editing screening, EdiGene advances the pipeline on therapeutic platforms of LEAPER-based in vivo RNA base editing, ex vivo gene-editing hematopoietic stem cells, and ex vivo gene-editing T cells for allogeneic CAR-T.

At Editas Medicine, we are pioneering the possible. Our mission and commitment is to harness the power and potential of CRISPR gene editing to develop a robust pipeline of medicines for people living with serious diseases around the world. Our goal is to discover, develop, manufacture, and commercialize transformative, durable genomic medicines for many diseases.

Elpiscience is a clinical-stage biopharmaceutical company dedicated to developing life-changing immuno-oncology therapies for cancer patients worldwide. The company’s innovative approach is focused on removing immunosuppressive factors in the tumor microenvironment, by targeting the adenosine pathway and myeloid checkpoints. A pipeline of novel molecules has been developed using its proprietary platforms including a powerful Bispecific Macrophage Engager (BiME®) technology that connects and activates macrophages for solid tumor killing without causing cytokine storms.

Epic Bio is a biotechnology company that focuses on biomedical research and genetics.

Developing transformative cancer medicines with CD19-Redirected ARTEMIS T Cells to improve patients’ lives

HuidaGene Biotechnology Co., Ltd (辉大基因) is a global biotechnology company focusing on the discovering, engineering, and developing CRISPR-based genetic medicine to rewrite the future of genomic medicine. Based in Shanghai and New Jersey, HuidaGene is committed to addressing patient's needs globally with various preclinical therapeutic programs covering ophthalmology, otology, myology, and neurology. Company's CRISPR-based therapeutics offer the potential to cure patients with life-threatening conditions by repairing the cause of their disease. We are committed to transform the future of genome-editing medicine.

iECURE is a clinical-stage biotechnology company focused on delivering cures and transforming the lives of patients with devastating liver disorders, utilizing new advancements in genetic medicine. We are advancing our pipeline in close partnership with the world-class translational engine at the University of Pennsylvania’s Gene Therapy Program. Using in vivo editing, our methods focus on inserting functioning genes into patients’ genomes, which offers long-term, stable expression of those genes. With our team’s proven track record, as well as the University of Pennsylvania’s deep expertise and translational genetic medicine engine, reversing the course of these devastating diseases is now within reach.

Inari is the SEEDesign™ company, using new breeding technology to push the boundaries of what is possible by designing nature-positive seeds for a more sustainable global food system. A combination of AI-powered predictive design and a pioneered multiplex gene editing toolbox enables us to unlock the full potential of seed and advance critical solutions with broad applications for growing more food with fewer resources.

Intima Bioscience - A clinical stage gene and cell therapy company focused on curative intent in solid tumor cancer

Jacobio Pharma (1167.HK) is committed to developing and providing new and innovative products and solutions to improve people's health. Our pipeline revolves around novel molecular targets on six major signalling pathways: KRAS, immune checkpoints, tumor metabolism, P53, RB and MYC. We aim for our key projects to be among the top three in the world. Our vision is to become a global leader recognized for our impact in drug R&D together with our partners. Jacobio has R&D centers in Beijing, Shanghai and Boston with our Induced Allosteric Drug Discovery Platform (IADDP) and our iADC Platform.

Kamau Therapeutics is a clinical-stage, next-generation gene correction company harnessing high efficiency targeted gene integration to develop a new class of therapies with the aim to cure a wide range of serious and life-threatening diseases. Our novel gene correction approach, called homology-directed repair (HDR), overcomes prior limitations in specificity, efficiency, and durability of gene editing to offer broad potential for transforming human health outcomes through the delivery of one-time curative cell therapies. Our platform aims treat or cure a range of serious genetic diseases with unmet medical needs.

Korro Bio was founded to turn extraordinary scientific insights into life-altering new treatments for patients. We harness the body’s natural RNA editing machinery to make precise, single-base RNA edits to modulate protein function, addressing the medical needs of patients. Our mission is to rewrite the future for patients living with debilitating disease by repairing the underlying genetic cause.

KSQ Therapeutics has built an industry-leading genome-scale precision functional genomics platform, called CRISPRomics, to pinpoint therapeutic nodes that directly correlate genomic function to disease with unprecedented certainty and speed. The company is deploying CRISPRomics for novel drug development across broad therapeutic areas and is currently advancing a proprietary pipeline of tumor- and immune-focused drug candidates.

Next generation CRISPR technology for programmed cell death. Focused on developing therapies for infectious disease and the microbiome. Locus Biosciences’ novel approach to precision antimicrobials works by taking advantage of a part of an immune system present in many bacteria called the CRISPR-Cas system. The CRISPR-Cas system protects bacteria from invaders such as viruses by creating small strands of RNA called CRISPR RNAs, which match DNA sequences specific to a given invader. When the CRISPR RNAs find a match, they activate Cas proteins that cut the DNA.

Lyell Immunopharma, Inc., a T cell reprogramming company, engages in developing T cell therapies for patients with solid tumors. The company develops therapies using technology platforms, such as Gen-R, an ex vivo genetic reprogramming technology to overcome T cell exhaustion; and Epi-R, an ex vivo epigenetic reprogramming technology to generate population of T cells with durable stemness. Its pipeline includes LYL797, a T cell product candidate for the treatment of non-small cell lung cancer and triple negative breast cancers; LYL845 for multiple solid tumors; and NY-ESO-1 for synovial sarcoma and other solid tumor indications. Lyell Immunopharma, Inc. was incorporated in 2018 and is headquartered in South San Francisco, California.

March Biosciences, Inc is a clinical-stage cell therapy company with a mission to transform patient care in the most challenging hematological malignancies. March Bio's lead asset is at the end of Phase I trials for T-cell malignancies, diseases with a dismal prognosis that is currently unaddressed by CAR-T and targeted therapy technologies. The company's pipeline is designed to expand on these early successes to more broadly impact other high-challenge cancers.

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.

Multitude Therapeutics (MTX) is a spinoff company created by Abmart to leverage its unparalleled target and antibody discovery capability for cancer immunotherapy. MTX focuses on developing novel antibody-drug conjugates (ADC), CAR-Ts and immuno-oncology antibodies for solid and hematological tumors. MTX's therapeutic programs are based on novel cancer and immune cell surface targets discovered by monoclonal antibody arrays (Human Membrane Proteome MabArrayTM or HMP-MabArrayTM) powered by the exceptional antibody discovery capabilities built at Abmart.

Obsidian Therapeutics is pioneering controllable cell and gene therapies to deliver transformative outcomes for patients with intractable diseases. Obsidian’s programs apply our cytoDriveTM technology in cell and gene therapy products to control expression of proteins for enhanced therapeutic efficacy, including our lead program cytoTIL15 engineered to make TILs more effective for more patients. Our aspirations are high, and we’re built to deliver – well-funded with blue chip investors, engaged field-leading advisors, strong partnerships with industry leaders, and a highly experienced, dynamic, innovative and collaborative team, collectively focused on delivering transformative therapies in areas of greatest clinical need. Located in the heart of Cambridge with a new laboratory facility in Bedford, we’re proud of our diverse talented team and committed to cultivating an environment of inclusion where we strive to instill a strong sense of belonging, and support each Obsidianite to continuously learn and contribute their best work. We offer competitive salary and benefits, and potential for employee ownership through stock options.

Prime Medicine was founded to bring the promise of gene editing to patients. We use Prime Editing, a next-generation technology that can “search and replace” to restore normal genetic function almost anywhere in the genome. Prime Editing searches to find the precise place in the genome to edit and replaces the segment of faulty DNA with a correct copy of DNA. A single Prime Editor can correct the individual mutations found across patients, meaning Prime Editing can potentially address more than 90 percent of known disease-causing genetic mutations. We envision a world where this technology can cure, halt, and ultimately prevent genetic diseases, providing lifelong benefit to patients. Prime Medicine offers the opportunity to shape the future of gene editing and transform how medicines are used to treat disease. Realizing the promise of Prime Editing requires a talented team with diversity of viewpoints and expertise. We’re building a group of dedicated, scientifically curious individuals who are passionate about helping patients live longer, healthier lives.

At Promega Corporation, creativity and connection drive discovery and innovation. We celebrate scientific discovery and the creative application of science to solve problems. Interactions with our customers, partners and vendors are central to everything we do, and we value those relationships. We seek out and build connections across the globe and in our local communities because creative problem solving requires a network of diverse ideas and viewpoints. We develop and evolve technologies and instrumentation in collaboration with scientists and researchers around the globe. From simple cloning vectors used to address fundamental questions in biology to bioluminescent assays that help us monitor food safety and water quality, we work side-by-side with our partners in academic, industrial and government institutions to bring the best minds to bear on the toughest questions. Since 1978, we have done this all with a steadfast commitment to sustainable business models that protect our environment, inspire and develop our employees as individuals, support the wellbeing and growth of our communities, and strengthen our relationships with our customers. With 16 branches around the world, Promega products are used by a global community of life scientists who are asking fundamental questions about biological processes as well as by scientists who are applying scientific knowledge to diagnose and treat diseases, discover new therapeutics, and use genetics and DNA testing for human identification.

Repare Therapeutics is a leading clinical-stage precision oncology company enabled by our proprietary synthetic lethality approach to discovering and developing novel therapeutics. We use our genome-wide, CRISPR-enabled SNIPRx® platform to systematically discover and develop highly targeted cancer therapies focused on genomic instability, including DNA damage repair. Our company was founded in 2016 by field-leading academics to systematically employ synthetic lethality insights and platforms and develop new precision oncology medicines. For more information, please visit www.reparerx.com. Scam Prevention Notice It has come to our attention that individuals posing as representatives of Repare Therapeutics have been engaging in fraudulent recruiting activities, offering fake job opportunities to unsuspecting candidates. These scams are designed to deceive job seekers into providing personal information or financial details under false pretenses. Please note that we will only contact you about a position that is posted on our careers page and official email communications from Repare employees will only come from a @reparerx.com email. Repare has a formal process which includes candidate application through the Repare career page (https://www.reparerx.com/careers/). If the job is not posted on the official Repare Therapeutics careers page, then it is not a legitimate position. If you have any questions or concerns regarding job opportunities at Repare Therapeutics, please don't hesitate to reach out to our HR department directly via Contact Us section (https://www.reparerx.com/contact/). Thank you for your vigilance and understanding.

Rocket Pharmaceuticals (NASDAQ: RCKT), Ltd is a biotechnology company focused on seeking cures through molecular and gene therapy. We are developing first-in-class treatments for patients with rare and often devastating orphan diseases using a lentiviral-based and adeno-associated virus (AAV) therapy approach. We have developed rewarding partnerships with a number of leading international research institutions as well as with centers of manufacturing and cell processing expertise, while building an internal team to drive the research, development, clinical and regulatory programs. Ultimately we aim to launch and globally commercialize innovative gene therapies in disease areas with high unmet need.

Sangamo Therapeutics is a genomic medicine company conducting research & development across four distinct but complementary technology platforms – gene therapy, cell therapy, in vivo genome editing, and in vivo genome regulation. The optionality and diversity inherent to Sangamo’s technology platform enables us to design therapeutic approaches to resolve the underlying genetic causes of disease, using whichever technology is best suited to deliver that treatment. Because Sangamo’s technology platforms are related, we are applying learnings across studies to optimize each individual platform and uncover research and development paths forward. Sangamo is best known for developing our proprietary gene editing technology, zinc finger nucleases (ZFNs). Our technology team continues to optimize ZFNs for the three key dimensions of genome editing: precision, efficiency, and specificity. Ex vivo gene-edited cell therapy is the most straightforward application of ZFN technology. It builds on early experience with HIV clinical studies and is applied to hematological disorders and oncology. Our cell therapy portfolio also includes ex vivo engineered regulatory T-cells (CAR-Tregs) for application in autoimmunity and inflammation. The insights gained about molecular biology, cassette engineering, and delivery through in vivo application of ZFNs led to our gene therapy program using AAV, which is advancing in the clinic. Zinc finger protein engineering and AAV delivery are also foundational for our zinc finger protein-transcription factor (ZFP-TF) genome regulation technology, which we are applying in CNS diseases. With our robust history of genomic medicine firsts, unmatched technology platform, and growing manufacturing and clinical development capabilities, Sangamo believes we are the company to realize the vision of genomic medicine.

SIMR is an innovation-oriented biopharmaceutical company dedicated to the discovery and development of new drugs for pain, inflammation and central nervous system related diseases. Since its establishment in 2014, the company has established a research and development platform of more than 3,000 square meters, with comprehensive drug development capabilities such as compound screening, CMC research, preclinical evaluation, and clinical development.

SNIPR Biome is the leading CRISPR Microbiome company. Our mission is to develop CRISPR-based medicines that give hope for effective and safe treatment of difficult-to-treat diseases in the future. Precision killing of bacteria has the impact to revolutionise the management of untreatable and difficult-to-treat infections as well as complex diseases directly impacted by the human microbiota.

SparingVision is a genomic medicines company, translating pioneering science into vision-saving treatments. Founded to advance over 20 years of world-leading ophthalmic research from its scientific founders at the Paris Vision Institute, SparingVision is leading a step shift in how ocular diseases are treated, moving beyond single gene correction therapies. At the heart of this is a pipeline of gene independent treatments for rod-cone dystrophies. Lead products, SPVN06 and SPVN20, address mid and late stages of retinitis pigmentosa (RP)respectively. RP is the most common inherited retinal disease affecting two million people worldwide. These novel medicines could form the basis of a suite of new sight-saving treatments with potential applications across many other retinal diseases, regardless of genetic cause. The Company is supported by a strong, internationally renowned team who aim to harness the potential of genomic medicine to deliver new treatments to all ocular disease patients as quickly as possible. SparingVision has raised €60 million to date and its investors include 4BIO Capital, Advent France Biotechnology, Bpifrance, Foundation Fighting Blindness (US), Fondation Voir & Entendre, UPMC Enterprises, Jeito Capital and Ysios Capital. For more information, please visit www.sparingvision.com.

SpliceBio is a biotechnology company exploiting Protein Splicing to develop the next generation of gene therapies. Our proprietary platform is based on technology developed in the Muir Lab at Princeton University after more than 20 years of pioneering intein and protein engineering research by our co-founders. The company is backed by UCB Ventures, Ysios Capital, NEA, Gilde Healthcare, Novartis Venture Fund and Asabys Partners.

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.

Tango Therapeutics is a biotechnology company discovering and developing novel medicines targeting cancer vulnerabilities to deliver transformational new therapies for patients. Tango was launched in 2017 with a $55 million Series A investment from Third Rock Ventures. The company has established a robust product engine that leverages advances in DNA sequencing and CRISPR-based target discovery to generate breakthrough medicines that have the potential to provide deeper, more sustained benefit than today’s targeted therapies, and extend the benefit of available immuno-oncology agents. Tango Therapeutics is focused on three areas of drug development, each in well-defined patient populations currently lacking effective treatment options, and each with hallmarks of cancer that have not been targeted yet. These include: loss of tumor suppressor gene function; multiple oncogenic drivers; and immune evasion. What fuels each of Tango’s programs is an increasingly sophisticated ability to utilize synthetic lethality - the interaction between two genes that causes cell death when both are inactivated. In cancer cells, one of these genes is inactivated by mutation; the other will be inactivated by a drug. This approach leaves normal cells largely unaffected, with the potential to greatly enhance anti-tumor efficacy and reduce associated toxicity. Tango’s success will be driven by its depth of understanding of the genetic subtypes of cancer, and corresponding insights into novel drug targets and combinations uniquely relevant to each subtype. By shaping discovery efforts in this way, Tango has the potential to reach the clinic quickly, and with a clear plan for identifying the patients most likely to benefit from each new treatment, an approach that could increase both speed and probability of success in translating novel target discoveries into transformational new medicines for patients.

TC BioPharm is a publicly traded, clinical-stage cell therapy company developing advanced allogeneic CAR-T cell therapy products for the treatment of cancer, as well as developing gamma delta T cell therapies for the treatment of infectious disease. We have a growing team based in our Glasgow office, alongside a wide range of scientific advisors, and partner organisations.

We are a cell therapy company that combines a novel patient engineering approach with targeted therapies to provide a solution for patients suffering from hematological malignancies.

We are leading a new era of precision medicine in which rationally designed oligonucleotides are the key to delivering safer, more effective medicines. These include potentially transformational therapies for conditions where there are currently few or no treatment options. We aspire to help create a world in which every patient with a genetic disease will have access to life-changing treatments in their lifetime.

Wugen is a clinical-stage biotechnology company developing the next generation of off-the-shelf memory natural killer (NK) and CAR-T cell therapies for cancer. Wugen is leveraging its proprietary Moneta platform and deep genomic engineering expertise to pioneer a new class of memory NK cell therapies to treat hematological and solid tumor malignancies. Wugen is based out of St. Louis, Missouri and San Diego, California.

YolTech Therapeutics is a biotech focuses on combining mRNA and gene editing technology to develop next-generation mRNA drugs and in vivo gene editing drugs.Our company is a pre-clinical stage gene editing biotech, has built leading genome editing platform and mRNA-LNP library. It possesses strong capability of novel Cas and base editors discovery and exceptional production capacity for LNP drug manufactory, with strong IP protection globally. It has created a pipeline with 10 genetic medicines focusing on cardiovascular diseases, infectious diseases and rare diseases.