Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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.
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).
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.
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).
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).
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.
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.
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.
Topline results from the Phase 2 EXACT clinical trial are expected in February 2023 with interim results in the second half of this year.
As part of the collaboration's expansion, ONO will contribute novel binding domains targeting a second solid tumor antigen.
NCI will work to generate proof of concept utilizing the Alaunos' proprietary non-viral Sleeping Beauty technology for personalized TCR-T cell therapy.
The positive CHMP recommendation could possibly make val-rox the first gene therapy for hemophilia A approved in Europe.
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.
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.