The American Society of Gene & Cell Therapy (ASGCT), a key professional membership organization for gene and cell therapy, published the Q3 edition of “Gene, Cell, & RNA Therapy Landscape,” a data report the organization launched earlier this year. Greater Philadelphia’s contributions to the cell and gene therapy space are well represented in the document.

A list of 19 gene therapies approved for clinical use includes three that originated from the Greater Philadelphia area:

  • Kymriah, originated by Novartis, was approved in 2017 to treat acute lymphocytic leukemia and diffuse large B-cell lymphoma.
  • Luxturna, originated by Spark Therapeutics (Roche), was approved in 2017 to treat Leber’s congenital amaurosis and retinitis pigmentosa.
  • Zolgensma, originated by Novartis, was approved in 2019 to treat spinal muscular atrophy.

The data report also covers an overview of dealmaking, referencing 139 alliance, acquisition, and financing deals announced in Q3 2021. Among those noted was Philadelphia-based Amicus Therapeutics, which spun out gene therapy assets into Caritas Therapeutics in September. They later reverse merged with ARTA Sciences Acquisition (SPAC) to gain a Nasdaq listing.

Also, in September, Malvern-based Ocugen licensed a therapy for age-related macular degeneration to CanSino Biologics Inc., and gene-editing firm iECURE raised $50M from Versant Ventures and Oribimed Advisors (source: DealForma).

According to the report, deals like these contribute to the astonishing number of gene and cell therapies in development globally, totaling 3,366. This figure includes technologies ranging from the preclinical stage through preregistration stages.

Greater Philadelphia area enterprises are contributing to this growth. During the past five years alone, according to DealForma, 32 regionally based enterprises have struck deals to out-license cell or gene therapy-related technologies. These discoveries are finding corporate homes regionally, across the U.S., and as far away as China, Israel, Japan, and Switzerland. For 26 of the 32, the headquarter city of the acquirer was identified. Among this group, 50% are located in Greater Philadelphia, evidence of the collaborative and connective attributes of the region.


ASGCT works to advance knowledge, awareness, and education leading to the discovery and clinical application of genetic and cellular therapies to alleviate human disease. Its membership includes scientists, physicians, patient advocates, and other professionals interested in gene and cell therapy.