List of Gene Therapy Companies in Pennsylvania - 51

Adaptimmune is a fully integrated cell therapy company, designed and built from the ground up with four U.K.- and U.S.-based biotechnology hub locations. Our comprehensive capabilities and teams include preclinical research, clinical development, translational sciences, autologous and allogeneic manufacturing, and in-house commercial and corporate operations. For more information about Adaptimmune, please visit www.adaptimmune.com. If interested in joining our fast growing team, click on the careers tab of this page.

Allegheny Health Network is an integrated health care delivery system serving the greater Western Pennsylvania region. More than 2,600 physicians and 22,000 employees serve the system's 14 hospitals as well as its ambulatory medical and surgery centers, Health + Wellness Pavilions, and hundreds of physician practice locations. AHN is a proud part of Highmark Health.

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.

AUM LifeTech is an American Biotechnology company focused on using next-generation RNA silencing technology to develop solutions in diverse life science verticals including biomedical research, medicine, agriculture, and aquaculture. Specifically, AUM's custom products include next generation of innovative genetic tools in the area of gene silencing and manipulation for biomedical research and therapeutic development. AUM’s non-GMO products are also being developed for broad-spectrum target-specific pest management and disease control in agriculture. RNA silencing products using FANA technology provide superior alternatives to conventional RNAi and gene editing approaches like CRISPR and provide a fast track target discovery. AUM's goal is to provide innovation at the genetic level for a better life.

From Discovery to Delivery™, Azzur Group provides the life science community full life-cycle solutions for all their GxP needs. From Azzur Cleanrooms on Demand™ facilities, to our labs, training centers and consulting offices across the nation, Azzur Group helps organizations start, scale, and sustain their growing enterprises. With nearly four decades of service to the life science community, we have become a trusted partner to the world's leading pharmaceutical, biotechnology, medical device, and healthcare companies, as well as their supply chain. Follow us on LinkedIn. For more information, visit azzur.com

A small, agile CRO. The primary function of the scientists at BioAnalysis, LLC is to provide a collaborative workspace that enables productive and effective solutions to the client’s problems. The traditional CRO model works well, however, at BioAnalysis, LLC we believe that collaborative interactions, communication and robust experimental design along with gold-standard quality results are the true value-added. The scientists of BioAnalysis, LLC possess a wealth of experience in characterization of therapeutic proteins, mAbs, and ADC (DARs), non-biologics (Polymers, PEO/PLA, nanoparticles), ICH/FDA regulatory applications, process development, and assessment of final biologic product formulations. Our subject matter expertise in the areas of Biophysics and Proteomics is showcased in over 70 peer reviewed publications.

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.

Cabaletta Bio, Inc., a clinical-stage biotechnology company, focuses on the discovery and development of engineered T cell therapies for patients with B cell-mediated autoimmune diseases. Its proprietary technology utilizes chimeric autoantibody receptor (CAAR) T cells that are designed to selectively bind and eliminate B cells, which produce disease-causing autoantibodies or pathogenic B cells. The company’s lead product candidate is DSG3-CAART, which is in Phase I clinical trial for the treatment of mucosal pemphigus vulgaris, an autoimmune blistering skin disease, and Hemophilia A with Factor VIII alloantibodies. Its product candidate pipeline also include MuSK-CAART, a preclinical stage product to treat a subset of patients with myasthenia gravis; FVIII-CAART, a discovery stage product to treat a subset of patients with Hemophilia A; and DSG3/1-CAART, a discovery stage product for the treatment of mucocutaneous pemphigus vulgaris. Cabaletta Bio, Inc. has a collaboration with the University of Pennsylvania; and research agreement with The Regents of the University of California. The company was formerly known as Tycho Therapeutics, Inc. and changed its name to Cabaletta Bio, Inc. in August 2018. Cabaletta Bio, Inc. was incorporated in 2017 and is headquartered in Philadelphia, Pennsylvania.

Carisma Therapeutics Inc. is a clinical stage biopharmaceutical company focused on utilizing our proprietary macrophage and monocyte cell engineering platform to develop transformative immunotherapies to treat cancer and other serious diseases. We have created a comprehensive, differentiated proprietary cell therapy platform focused on engineered macrophages and monocytes, cells that play a crucial role in both the innate and adaptive immune response. Carisma is headquartered in Philadelphia, PA.

Carnegie Mellon University is a Higher Education institution that focuses on research and technology innovation.

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.

Center for Breakthrough Medicines is an innovative cell and gene therapy organization focused on contract development and manufacturing organization located in the heart of “Cellicon Valley.”

Century Therapeutics, Inc., a biotechnology company, develops transformative allogeneic cell therapies for the treatment of solid tumor and hematological malignancies. The company’s lead product candidate is CNTY-101, an allogeneic, induced pluripotent stem cells (iPSCs)-derived chimeric antigen receptors (CAR)-iNK cell therapy targeting CD19 for relapsed, refractory B-cell lymphoma. It is also developing CNTY-103, a CAR-iNK candidate targeting CD133 + EGFR for recurrent glioblastoma; CNTY-102, a CAR-iT or CAR-iNK candidate targeting CD19 + CD79b for relapsed, refractory B-cell lymphoma and other B-cell malignancies; and CNTY-104, a CAR-iT or CAR-iNK multi-specific candidate for acute myeloid leukemia. Century Therapeutics, Inc. has a research collaboration with Outpace Bio, Inc. for CAR engineering. The company was founded in 2018 and is headquartered in Philadelphia, Pennsylvania.

Chimeron Bio is a biotechnology company focused on developing personalized gene therapies, including self-amplifying RNA vaccines and therapeutics designed on its proprietary technology.

The future of single-use, column-free purification is here. We are dedicated to empowering biopharmaceutical manufacturers with our cutting-edge, columnless single-use continuous chromatography platform, offering enhanced flexibility, scalability, and purity, reducing downtime and resin consumption, and maximizing productivity for the cost-effective production of life-saving therapies. With more efficient process development, clinical manufacturing, and commercial manufacturing, we aim to help our customers provide the most affordable therapeutics possible to their patients. Join us for our virtual ChromaChats™ roundtables to discuss and solve challenges in the field of chromatography with other leaders, network with their peers, collaborate on projects, share successes and pitfalls, and connect with other leaders. Learn more here: https://chromatan.com/chromachats/

Code Bio is developing a transformational, new class of precision genetic medicines to treat life-threatening diseases.

Coeptis Therapeutics Holdings, Inc. (NASDAQ: COEP) is a biopharmaceutical company developing innovative cell therapy platforms for patients with cancer.

CSL Behring is a biopharmaceutical company that manufactures plasma-derived and recombinant therapeutic products.COVID-19: CSL says it is working on a plasma-based therapy for patients with more severe forms of COVID-19.The company is part of the CSL family of companies. The parent company, CSL Limited is based in Melbourne, Australia, employs more than 30,000 people, and delivers its life-saving therapies to people in more than 100 countries.

GEMMABio, a new therapeutics company, will serve as the research and innovation arm to speed the research of and global access to life-changing advanced therapies for those living with rare diseases. Alongside Franklin Biolabs, GEMMABio will advance gene therapy research and deliver therapeutics to patients around the world significantly faster.

GeneLancet Biosciences is a gene editing company offering high-quality ligated single-molecule guide RNAs (LgRNA) and highly-reliable and cost-effective arrayed LgRNA libraries.

At Geneos, our passion is to develop personalized therapies to unleash the most powerful force against cancer – your body’s own immune system. Our GT-EPIC Platform is designed to identify relevant neoantigen targets and then design, manufacture, and deliver tumor specific neoantigen-targeted personalized immunotherapies.

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.

INOVIO is a biotechnology company committed to powering a new way forward in DNA medicines to save and protect lives worldwide. The company is focused on rapidly bringing to market precisely designed and delivered DNA medicines to potentially treat and protect people from serious and life-threatening diseases associated with HPV, cancer, and infectious diseases.

Interius is an early-stage biotechnology company expanding the potential of cell and gene therapy by developing next-generation delivery technology based on inventions from the laboratory of Dr. Saar Gill at the University of Pennsylvania.

IPS-Integrated Project Services, LLC, a Berkshire Hathaway company, is a global leader in developing innovative solutions for the consulting, architecture, engineering, project controls, procurement, construction management, and CQV of technically complex facilities. With technical expertise spanning R&D to pilot-scale to large-scale production, our team specializes in the technology, trends, and regulatory environment to successfully deliver capital projects and improve operations that enable out clients to deliver life-impacting products worldwide. Headquartered in Pennsylvania, IPS is a multinational company with professionals and offices in the United States, Canada, Brazil, the United Kingdom, Ireland, Germany, Denmark, Switzerland, Australia, Singapore, China, and India. With the acquisition of Linesight, IPS has over 3,000 professionals in 45+ offices across 17 countries. Linesight specializes in cost, schedule, risk, program, and project management services in various market sectors, including data centers, life sciences, and high-tech industrial.

The Jefferson Institute for Bioprocessing (JIB) offers a broad range of trainings to advance the skills and knowledge of scientists, engineers, and technicians who work in process development and biomanufacturing of biopharmaceuticals and biologics. Through its 25,000 sq. ft. fully flexible state-of-the-art facility, JIB is able to provide truly tactile training by combining interactive presentations, workshops, hands-on lab and pilot-scale experience. JIB offers customized trainings to meet industry needs by providing specialized courses developed through face-to face planning and delivered either at JIB or at the company site. JIB is the only education and training institute for biopharmaceutical processing in North America that combines a commercial scale GE FLEX Factory with degree granting programs through Thomas Jefferson University, customized training courses and bioprocess solutions to meet the needs of your company.

KaliVir Immunotherapeutics was founded in 2019 to pioneer a unique and novel approach to cancer treatment that engages a virus' natural ability to replicate in and kill cancer cells. This modality is known as oncolytic viral immunotherapy. KaliVir's cutting-edge, next generation oncolytic viral immunotherapy programs combine technologies developed in-house and licensed from University of Pittsburgh's world-class oncolytic virus and immunotherapy research programs.

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)

At Latus Bio, we are developing novel therapeutics for CNS disorders with our revolutionary AAV capsids. We have developed capsid variants with unprecedented potency, specificity, and trophism. All of our capsids were designed with a particular disease in mind, in order to find the right capsid and the right route of administration to target the most relevant brain regions and cell types for each specific indication. Founded on the groundbreaking work of Professor Beverly Davidson from the Children's Hospital of Philadelphia, Latus Bio is committed to transforming patient care through targeted, potent, and safe gene therapies. We are focused on CNS diseases such as CLN2 (Batten Disease) and Huntington’s Disease, aiming to fundamentally alter the course of these challenging conditions by always putting the patients first. We are not just developing therapies; we are revolutionizing the future of healthcare with innovations that reduce therapeutic load, enhance safety, facilitate manufacturing, and expand access. Latus Bio is powered by a diverse team of visionary scientists, clinicians, and industry leaders dedicated to tackling some of the most complex neurological disorders. With a robust pipeline and a strategy to enter clinical trials by late 2025, we are poised to make significant strides quickly. Be part of this transformative movement at Latus Bio. Let's unlock the potential of gene therapy to bring hope and healing to millions around the globe. Learn more about our mission, our people, and our promise at www.latusbio.com and connect with us on LinkedIn to follow our progress.

NanoCell Therapeutics is a privately-held biotechnology company with offices in King of Prussia, Pennsylvania, and Utrecht, the Netherlands. Our mission is to revolutionize in-vivo cell engineering through our groundbreaking non-viral, DNA-based gene therapy platform, primarily focusing on oncology and autoimmune diseases. At NanoCell, we’re driving innovation by transforming traditional adoptive cell therapy methods into advanced in-vivo treatments. Our ultimate goal is to simplify the treatment process, increasing patient accessibility, potential clinical benefits, and cost-effective manufacturing. We work both autonomously and in collaboration with partners to make substantial advancements in our targeted disease areas. For more information, visit http://www.nanocelltx.com).

NeuExcell Therapeutics is a gene therapy company focusing on improving the lives of patients suffering from neurodegenerative diseases. We have discovered a disruptive technology that focuses on regenerating neurons in the brain. This powerful platform has the potential to transform patient outcomes and change lives. We are now rapidly expanding our R&D efforts, toward our goal of developing effective and safe treatments for serious neurodegenerative diseases including stroke, Huntington’s Disease, ALS, and Traumatic Brain Injury. Our experienced team is excited to dedicate themselves to contribute and bring innovative and effective treatments for those suffering from neurodegenerative conditions. It is our goal to improve the quality of life of millions of patients worldwide by using the power of gene therapy to restore damaged neuronal tissue.

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.

Passage Bio is a genetic medicines company based in Philadelphia, PA that is developing a deep pipeline of adeno-associated virus (AAV) delivered therapeutics for the treatment of rare monogenic central nervous system disorders (CNS). We seek to deliver transformative therapies to patients by leveraging our team’s decades of experience, including our founders, as well as the transformative potential genetic medicine technology to develop treatments that improve outcomes for patients with serious, life-threatening, rare CNS disorders.

Polysciences introduces Kyfora Bio, our bioprocessing brand, supporting development of cell and gene therapy (CGT) breakthroughs from bench to clinic. Backed by 60+ years of specialty chemical development, manufacturing, and application expertise, Kyfora provides research grade through cGMP transfection reagents and excipients for viral vectors, mRNA vaccines, and other drug delivery applications. Our cost-effective and scalable cGMP processes empower advanced therapies to improve patient lives.

Prolifagen Therapeutics is a biotech startup dedicated to the prevention of heart failure in patients with large myocardial infarcts. The company is focused on cardiac regeneration based on microRNA technology.

Puresyn, Inc. is focused on and dedicated to providing manufacturing services capable of producing high quality DNA which can be used for many applications including GMP manufacture of gene therapy products.

Rx-360 is a pharmaceutical industry consortium that aims to improve the quality and security of supply chains for proprietary and generic pharmaceutical companies and suppliers.

Scout’s mission is to harness the genetic revolution transforming human medicine to deliver the future of veterinary medicine. By combining world-leading talent in animal health and gene therapy technology, we have built a development platform for one-time therapies that address major areas of unmet medical need in pet medicine. We are a global company supported by leading life sciences investors and an R&D collaboration with the renowned Gene Therapy Program at the University of Pennsylvania. About Our Currently Enrolling Study for Cats Scout’s first therapy in development focuses on the anemia associated with chronic kidney disease (CKD) in cats. Our study is currently enrolling cats at participating clinics across the U.S. It involves a single injection (followed by 70 days of monitoring) and may relieve a cat’s anemia for life. Learn more about our study and find a participating clinic: www.scoutbio.co/catEPOstudy.

At Spark® Therapeutics, we believe in a world where no life is limited by disease.   We were born of innovation, springing from the curiosity, imagination and dedication of remarkable scientists and healthcare visionaries. Our mission is to unlock the power of gene therapy to accelerate healthcare transformation.     Since our founding, we have been committed to bringing a wide range of expertise to build a fully integrated gene therapy company focused on inherited retinal diseases (IRDs), neurodegenerative diseases, as well as diseases that can be addressed by targeting the liver, such as hemophilia. We are seeking talented individuals with diverse experiences, abilities and interests who have the curiosity, courage and drive to reimagine a new health care paradigm.     Join us on a journey through uncharted territory – seeking to bring gene therapy to people worldwide. The resilience of the people we serve is our inspiration to break barriers, as we strive to turn genes into medicine. Spark® is a member of the Roche Group.     Know that working at Spark Therapeutics is not just another job; it is a once-in-a-lifetime opportunity. We embrace the challenges before us and the uncertainty inherent in them. Ultimately, we are working to create a world where no life is limited by disease. To learn more about Spark and our open positions, visit www.sparktx.com. You can also find us on Twitter at @Spark_tx.  Spark’s social media sites are monitored from Monday to Friday during normal business hours. Questions or concerns should be directed here: https://sparktx.com/contact-us/.

Acquired by Sumitomo Dainippon Pharma of Japan. Spirovant Sciences is a gene therapy company focused on changing the course of cystic fibrosis and other respiratory diseases. The company's current focus is on developing novel gene therapies for cystic fibrosis and other pulmonary diseases. Spirovant is located in Philadelphia, PA's thriving Life Sciences Corridor and has a diverse management team, researchers, and partners.

Temple University is a higher education institution that offers a wide range of academic programs and degrees.

The Wistar Institute is a biomedical research institution that focuses on advancing knowledge in life sciences and medical research.

A privately held company, since 1996 ValSource has remained the better solution for the regulated pharmaceutical industry worldwide. The ValSource family of companies delivers Line of Sight approach consulting services, resources and solutions for the biological, medical device, and pharmaceutical industries. The Line of Sight approach begins with the end objective in mind. It links critical phases of process development, validation, and manufacturing, doing what is necessary, but only what is necessary, to ensure effective, compliant processes. By process mapping from the desired outcome to the process start, we logically develop better objective-focused solutions. ValSource’s 300+ strong talent force is comprised of full-time employees. We are mechanical engineers, electrical engineers, computer engineers, computer scientists, chemical engineers, chemists, statisticians, life scientists and microbiologists. As industry leaders, we serve in positions of consequence, using critical thinking to help shape the conversation and drive progress for our industry and the clients we serve.

Vector Biolabs, a Fortis Life Sciences Company, is focused on developing robust gene delivery products through use of proprietary tools and technologies. Vector has been the leading CMO in research, discovery, and preclinical viral vector production for over 20 years, and has constructed and produced more than thirty thousand AAVs and adenovirus capsids.

Verismo Therapeutics is pioneering the development of novel chimeric antigen receptors (CARs) that enhance T-cell-based cellular immunotherapy for cancer, especially non-hematologic malignancies with few effective therapeutic options. Verismo Therapeutic’s core technology is built upon CARs that are uniquely engineered to more realistically reflect the natural multichain design of killer immunoglobulin-like receptors (KIRs), an important family of immunologic receptors used by T cells and NK cells. Preclinical studies show that SynKIR™-T cells, created by introducing these KIR-based CARs (KIR-CARs) into T cells, more effectively eliminate aggressive tumors in vivo when compared with T cells bearing traditional 2nd generation CAR designs found in current FDA approved CAR-T cell therapies.

Villanova University is a higher education institution that offers undergraduate, graduate, and doctoral programs in various fields of study.

VintaBio’s development and manufacturing team has over a century of experience in the cell and gene therapy space and its members have played key roles in the development of the most advanced therapies on the market today. Located in the historic Navy Yards of Philadelphia – the epicenter of cell and gene therapy innovation – VintaBio specializes in developing and manufacturing consistent, high-quality viral vectors, a vital and currently underserved area of CGT development. The company was founded by Junwei Sun and Dr. Shangzhen Zhou, who led development of the AAV vectors behind the first two FDA-approved gene therapies, as well as the lentiviral vector for the first FDA-approved cell therapy. VintaBio has raised $64 million in venture funding, which has been used to develop a brand new 22,500 square foot facility in Philadelphia created specifically to facilitate efficient cell and gene therapy manufacturing.

WuXi Advanced Therapies - Accelerating Progress and Time to Market WuXi Advanced Therapies is a cell and gene therapy Contract Testing, Development and Manufacturing Organization (CTDMO) that is reducing the complexities of manufacturing by providing integrated platforms that enable cell and gene therapies to be developed, manufactured, and released faster and with greater predictability. Our Story By leveraging platforms and integrated testing our team provides expedited development and commercialization of cell, gene, protein and viral-based therapies. This approach enables new biopharmaceuticals to be developed, manufactured and released faster and with greater predictability, thereby reducing the complexities of high-touch, multi-vendor production models. Our solutions help clients overcome challenges to commercialization, including process development, manufacturing capacity, analytical development, and raw materials management. Multiple, scale-able, enabling platforms integrate manufacturing, process development and testing capabilities to provide greater predictability and speed to clinic. We support advanced therapy programs with extensive infrastructure and 400,000+ square feet of state-of-the-art, GMP-compliant facilities on our modern campus located at the Navy Yard in Philadelphia, Pennsylvania.

XyloCor Therapeutics is a development-stage, biotechnology company dedicated to developing first-in-class gene therapies for patients with unmet medical need in cardiovascular disease.