Around the Helix: Cell and Gene Therapy Company Updates – June 29, 2022

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.

1. Liso-Cel Approved for Second-Line Large B-Cell Lymphoma

The FDA has approved Bristol Myers Squibb’s supplemental biologics license application for lisocabtagene maraleucel (liso-cel; Breyanzi) as second-line therapy for patients with large B-cell lymphoma (LBCL).

2. Late-Onset Pompe Disease Gene Therapy Trial Placed on Clinical Hold

The FDA has placed a clinical hold on Astellas Pharma’s phase 1/2 FORTIS study (NCT04174105) evaluating the AT845 gene-replacement therapy for the potential treatment of late-onset Pompe disease (LOPD) after the incidence of a serious adverse event (AE) of peripheral sensory neuropathy.

3. Sarepta’s Duchenne Muscular Dystrophy Gene Therapy Placed on Clinical Hold

The FDA has placed a clinical hold on SRP-5051 (vesleteplirsen), Sarepta Therapeutics’ next-generation peptide-conjugated phosphorodiamidate morpholino oligomer, which is intended for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 51 skipping. The hold is related to a serious adverse event of hypomagnesemia that occurred in part B of the ongoing 2-part, phase 2 MOMENTUM clinical trial (NCT04004065).

4. Orphan Drug Designation Granted to MB-106 in Waldenstrom Macroglobulinemia

The FDA has granted orphan drug designation to MB-106, Mustang Bio’s CD20-targeted autologous chimeric antigen receptor (CAR) T-cell therapy for the treatment of Waldenstrom macroglobulinemia (WM).

5. Cell and Gene Therapy Experts Added to Advisory Board for Ambys Medicines

Ambys Medicines announced the formation of its clinical and scientific advisory boards which will include scientists pioneering cell and gene technologies in addition to leading clinical experts in liver disease.

6. Ocugen Receives Gene Therapy Patent

U.S. Patent No. 11,351,2258 has been issued to Ocugen which covers the use of a nuclear hormone receptor gene in treating and preventing retinal degenerative diseases.

7. Ixaka, Minaris Regenerative Medicine Partner on Chronic Limb Ischemia Cell Therapy

The companies have signed an agreement to enable technology transfer as well as clinical and commercial manufacturing of REX-001, Ixaka’s lead autologous multi-cell therapy being developed for the treatment of chronic limb-threatening ischemia in patients with diabetes.

8. XyloCor Reaches Target Enrollment in Phase 2 Gene Therapy Trial for Refractory Angina

Topline results from the Phase 2 EXACT clinical trial are expected in February 2023 with interim results in the second half of this year.

9. Fate, ONO Expand Collaboration for Allogeneic CAR NK and CAR-T Therapies

As part of the collaboration's expansion, ONO will contribute novel binding domains targeting a second solid tumor antigen.

10. Alaunos, NCI Extend R&D Agreement for Personalized TCR T-Cell Therapies

NCI will work to generate proof of concept utilizing the Alaunos' proprietary non-viral Sleeping Beauty technology for personalized TCR-T cell therapy.

11. EMA Puts Support Behind BioMarin's Hemophilia A Gene Therapy

The positive CHMP recommendation could possibly make val-rox the first gene therapy for hemophilia A approved in Europe.

12. Adverum Gets PRIME Designation for Wet AMD Gene Therapy

Adverum’s lead gene therapy candidate, ADVM-022 is a one-time, intravitreal injection for the treatment of patients with wet age-related macular degeneration.

13. BioNTech Wins PRIME Designation for Testicular Cancer CAR-T Candidate

The designation follows positive interim Phase 1/2 data for BNT211 that showed a good safety profile and early signs of anti-tumor activity in patients with heavily pretreated testicular cancer.