Around the Helix: Cell and Gene Therapy Company Updates – May 1, 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 Fidanacogene Elaparvovec-dzkt for Moderate to Severe Hemophilia B

The FDA has approved fidanacogene elaparvovec-dzkt (Beqvez; Pfizer) for the treatment of adults with moderate to severe hemophilia B who currently use factor IX prophylaxis therapy; or who have a history of, or current, life-threatening hemorrhage; or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid determined by an FDA-approved test.

2. Taysha Transfers Deprioritized Gene Therapy Programs Back to Partners

Taysha Gene Therapies is working on transferring deprioritized programs to collaborators, including its TSHA-120 AAV vector gene therapy program for giant axonal neuropathy.

3. NKGen’s NK Cell Therapy SNK01 Cleared for US Clinical Trial in Parkinson Disease

NKGen’s autologous natural killer (NK) cell therapy SNK01 has received clearance of an investigational new drug application from the FDA for a phase 1/2a clinical trial in patients with Parkinson disease.

4. Trial for Regeneration Biomedical’s Alzheimer Disease Stem Cell Therapy Doses First Patient

The first patient has been dosed in Regeneration Biomedical’s phase 1 clinical trial (NCT05667649) for RB-ADSC, an autologous, Wnt-activated adipose-derived stem cell therapy, for the treatment of Alzheimer Disease.

5. Regeneron and Mammoth Team Up for CRISPR-Focused Endeavor

Under a new collaboration agreement, the 2 companies will put their respective expertise together to pursue the development of in-vivo CRISPR-based gene therapies that will be targeted at a variety of tissue and cell types outside the liver, with the intention of treating a range of diseases.

6. Cullinan Seeks to Compete Against CAR-T in Autoimmune Disease With Its T-cell Engager

Cullinan Therapeutics, which was formerly known as Cullinan Oncology, has announced its intention to bring CLN-978, its half-life extended CD19xCD3 bispecific T-cell engager construct that was originally developed for B-cell nonHodgkin lymphoma, to autoimmune disease indications. Its first target in the new field will be systemic lupus erythematosus.

7. Mustang Bio Makes Public Offering

The chimeric antigen receptor T-cell (CAR-T)-focused biopharmaceutical company has announced a public offering of an aggregate of 16,877,638 shares of common stock, expected to gross around $4 million prior to deduction of placement agent's fees and other offering expenses. Notably, the company also recently announced its intention to bring one of its CAR-T products from oncology to autoimmune disease.

8. SalioGen Puts Forth SGT-1001 as Candidate for Stargardt Disease

SalioGen Therapeutics has announced its intention to develop its preclinical genetic medicine product SGT-1001, which uses the company's Gene Coding™ technology, for the treatment of ABCA4-mediated Stargardt disease. “The nomination of SalioGen’s first development candidate, SGT-1001, demonstrates many of the unique capabilities of our novel Gene Coding technology, including the ability to seamlessly integrate a gene as large as ABCA4 into the retina with a proprietary lipid nanoparticle,” Jason Cole, JD, the CEO of SalioGen Therapeutics, said in a statement.