Around the Helix: Cell and Gene Therapy Company Updates – August 7, 2024

The cell and gene therapy sectors are growing exponentially, with new players emerging daily and much progress being made both in and out of the lab. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.

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1. FDA Approves Adaptimmune’s TCR T-cell Therapy Afami-cel for Synovial Sarcoma

The FDA has granted accerlated approval to Adaptimmune Therapeutics’ afamitresgene autoleucel (afami-cel, formerly ADP-A2M4), an investigational T-cell receptor (TCR) T-cell therapy marketed as Tecelra, for the treatment of synovial sarcoma.

2. Galapagos Pauses Enrollment in Multiple Myeloma CAR-T Trial After Parkinsonism Case

Galapagos has paused enrollment in its phase 1/2 PAPILIO-1study evaluating GLPG5301 chimeric antigen receptor (CAR) T-cell therapy in patients with relapsed/refractory multiple myeloma after at least 2 prior lines of therapy after a patient developed a case of Parkinsonism.

3. FDA Modifies CAR-T Risk Strategies to Lower Healthcare Burden

The FDA has modified its Risk Evaluation and Mitigation Strategies for autologous CAR T-cell immunotherapies to minimize burdens on healthcare systems.

4. Umoja Biopharma's In Situ CAR-T Therapy Cleared for US Trial in Hematologic Malignancies

The FDA has cleared an investigational new drug application for Umoja Biopharma's UB-VV111, a gene therapy product intended to create CD19-directed CAR T-cells within the body, allowing the company to go forward with a phase 1 dose escalation trial in hematologic malignancies.

5. GEMMA Biotherapeutics Purchases Lysosomal Disease Gene Therapy Program Licenses From Passage Bio

The 3 out-licensed programs include gene therapies for GM1 gangliosidosis, Krabbe disease, and metachromatic leukodystrophy. In addition, the 2 companies have entered into a strategic research and collaboration agreement focused on the development of genetic medicines for several central nervous system diseases, including Huntington disease.

6. Precigen Lets Go of Staff and Rearranges Priorities

Precigen has decided to put its focus on advancing PRGN-2012 AdenoVerse gene therapy, its gene therapy product for the treatment of recurrent respiratory papillomatosis, through potential commercialization. As a result, the company has paused several of its UltraCAR-T clinical programs and all preclinical programs. Furthermore, the company is letting go more than 20% of its employees.

7. BioMarin Limits Geographic Scope of its Plans for Hemophilia A Gene Therapy

BioMarin has announced that it will be scaling back its efforts with regard to valoctocogene roxaparvovec (val-rox, marketed as Roctavian), its gene therapy product for the treatment of adults with severe hemophilia A. Going forward, the company is mainly focused on distributing the gene therapy in the United States, Germany, and Italy, 3 countries in which it has been approved for use by relevant regulatory authorities.

8. Patient in Abu Dhabi is First to Receive Muscular Dystrophy Gene Therapy Elevidys Outside the US

A patient treated at the Sheikh Khalifa Medical City in Abu Dhabi, United, is the first patient to have been treated with Sarepta Therapeutics’ delandistrogene moxeparvovec-rokl (marketed as Elevidys), an adeno-associated virus vector-based gene therapy for patients with Duchenne muscular dystrophy, outside the US.