There are 30 gene-therapy development companies in the Philadelphia area that have raised well over $1 billion in invested capital, and together employ more than 3,000 people. And that's not counting the principal scientists, doctors, and techs who cooperate with them at labs at the University of Pennsylvania, Jefferson, Children's Hospital of Philadelphia, and other teaching hospitals.
Penn was a cradle of gene-therapy experiments in the 1990s, and recent Food and Drug Administration approvals for commercial applications have attracted a flood of investors backing projects developed by faculty and staff. Penn trustees' vote to invest $50 million of university funds in faculty companies in April sparked "over $10 in co-investment for every dollar they invest," said Richard W. Vague, the marketing mogul-turned-investor and philanthropist, who endowed pioneering cellular immunologist and leukemia-fighter Carl June's faculty seat at Penn's Perelman School of Medicine.
The list shows how this region is poised to benefit as treatments advance to market and enrich their owners or are bought up by larger biopharma companies.
Many of the firms here are new companies founded in the last few years, backed by a wave of investors from the big biotech capital centers of Massachusetts, California, and China, as well as Penn and a handful of local capitalists. Other firms on the list date to Philadelphia's long history as a drug center and have adapted to new gene-therapy markets and technologies.
Philadelphia-area gene therapy companies
Here are large and small area gene therapy employers, listed alphabetically, as compiled by Holly Flanagan at Gabriel Investments, a firm Vague founded, with updated information for the largest area employer in the group, WuXi AppTec:
1. ACF Bioservices (subsidiary of Absorption Systems LP), Exton 1996
Local employees: 10
Analytical support for gene and cell therapy products
2. Abzyme Therapeutic, Royersford
Modular single domain VHH antibody fragments for immunotherapy, using proprietary antibody generation platforms.
3. Adaptimmune, Philadelphia — Navy Yard, 1993 (local since 2008)
Cancer immunotherapy: affinity enhanced T-cell receptor
Funding includes: $104 million: NEA, OrbiMed Advisors LLC, Wellington Management Company LLP, Fidelity Biosciences, Foresite Capital Management, Ridgeback Capital Management, Novo A/S, QVT, Rock Springs Capital, venBio Select, Merlin Nexus, University of Oxford; PLUS $176 million in 2015 IPO
4. Aevi Genomic Medicine (formerly Medgenics), Wayne, 2000
Translating genetic discoveries into novel therapies.
Funding includes: $78.5 million from Koor, Alta, Alta Berkeley
5. AUM LifeTech, Philadelphia
Gene silencing technology
6. Cabaletta Bio, Philadelphia
CAAR-T, an improved version of CAR-T
Funding: $38 million
7. Carisma Therapeutics, Philadelphia. 2016
Pioneer developer of CAR macrophages, a disruptive approach to immunotherapy
Funding includes: $53 million from HealthCap, AbbVie Biotech Ventures, Grazia Equity, IP Group Inc., Wellington Partners, TPG Biotech, MRL Ventures Fund, Agent Capital, Penn Medicine
8. Excision BioTherapeutics, Philadelphia, 2015
Funding includes: $10 million from Oakhouse, Artis Ventures
9. Fibrocell Science, Exton, 1992 (formerly Isolagen)
Autologous cell-therapy company developing products for aesthetic, medical, and scientific applications
Funding includes: $115M from 2009 IPO and share sales
10. FlowMetric Inc., Doylestown (Pennsylvania Biotech Center), 2010
Flow cytometry and cell-sorting services
Funding: $3.3 million
11. Formula Pharmaceuticals, Berwyn, 2010
Developing a proprietary, next-generation Chimeric Antigen Receptor (CAR) immunotherapy platform
12. Immunome, Exton, 2008
Funding includes: $21.7 million from BioAvance, Evolution
13. Immunotope, Doylestown (Pennsylvania Biotech Center) 2002
Immunotherapy for cancer and chronic viral infections
Funding includes: $1.5 million NIH, Ben Franklin Technology Partners
14. Imvax, Philadelphia (Chestnut Hill), 2015
Licenses Jefferson systemic cancer immunotherapy technology
Funding: $14.7 million
15. Inovio Pharma, Plymouth Meeting, 1983
Employees: 113 (total 278 including San Diego)
Researches, develops, commercializes synthetic vaccines, immune therapies for cancer and other infectious diseases.
Funding: $181 million. IPO in 1998
16. Lampire Biological Laboratories, Pipersville, 1988
Antibody, tissue culture, immunochemistry, blood products
17. Linnaeus Therapeutics, Haddonfield, 2015
Identification and development of small molecule agents that modulate cellular differentiation for the treatment of cancer and disorders of pigmentation.
Funding: $2.2 million
18. MBF Therapeutics, Ambler, 2008
Developing innovative gene-based checkpoint inhibitor and immune modulators for cancer and infectious diseases for the companion animal market
Funding: $1.1 million
19. Oncoceutics, Hummelstown, 2009
Developing a novel class of safe and effective cancer therapies called imipridones
Funding: Not disclosed, from Spring Mountain Capital
20. Perch Therapeutics, Philadelphia
21. Realm Therapeutics, Malvern, 2006
Funding: $26 million IPO 2017; previously OrbiMed, Abingworth, RA Capital, Polar Capital, Biotech Value Fund
22. Rockland Immunochemicals, Limerick, 1962
Manufacturing new tools for basic, applied and clinical research in functional genomics, gene therapy and drug discovery markets
23. Spark Therapeutics, Philadelphia (Navy Yard), 2011
Develops gene therapy products for the treatment of debilitating genetic diseases.
Funding: $122.8 million from IPO, earlier funders include CHOP, Sofinnova, Brookside Capital, Deerfield Management Company, Rock Springs Capital, undisclosed investors, and accounts managed by T. Rowe Price Associates, Inc., Wellington Management Company
24. Tmunity Therapeutics, Philadelphia (Center for Advanced Cellular Therapeutics), 2015
Employees: 41 (includes manufacturing lab in Norristown)
Cellular immunotherapies for the treatment of cancer.
Funding: $145 million from Penn Medicine, Lilly Asia Ventures, Parker Institute for Cancer Immunotherapy, Be The Match BioTherapies, Kleiner Perkins
25. Tycho Therapeutics, Philadelphia
Targeted therapy of autoimmunity
26. Virion Therapeutics, Newark, Delaware, 2018
Developing innovative immune-based treatments for virally-associated cancers. Started at Wistar Institute in Philadelphia.
Funding: Series A totalled $5.3 million (updated) from Robin Hood Ventures, Mid-Atlantic Bio Angels Group (MABA), Keiretsu Capital Fund III, Life Science Angels, Keiretsu Forum, Alliance of Angels, and Crimson Peak.
Plus these companies, not based in Philadelphia, but with significant operations here:
27. CSL Behring (Australia; U.S. HQ in King of Prussia)
Area employment: 500 (total 16,000 in 30 countries)
28. Arbutus Biopharma (Canada; U.S. HQ in Warminster)
Hepatitis B Virus product pipeline encompassing direct antiviral, host targeting and immune-based approaches
Total employment: 130
29. Immunocore (U.K.; U.S. offices in Conshohocken)
Discovery and development of novel T cell therapies
Total employment: 279 employees across both locations
Funding includes: $360 million from Woodford Investment Management, Malin Corporation plc, Eli Lilly and Company, RTW Investments
30. WuXi AppTec (Shanghai, China; Philadelphia facilities totaling 380,000 sf at the Navy Yard)
Area employment: 600
Gene and cell therapy contract development and management organization. Integrates contract development, manufacturing and testing, from pre-clinical to commercial, for commercial and academic clients, including Penn.