List of Adeno-associated viral vector (AAV) Companies in Pennsylvania - 22

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.

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

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.

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.

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.

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.

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.

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.

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.

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.

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.