Around the Helix: Cell and Gene Therapy Company Updates – January 17, 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. Vertex Nets Early Casgevy Approval for Transfusion-Dependent Thalassemia

The FDA has approved Vertex Pharma and CRISPR Therapeutics’ exagamglogene autotemcel (exa-cel; Casgevy) for the treatment of patients 12 years and older with transfusion-dependent beta thalassemia, over 2 months before the scheduled March Prescription Drug User Fee Act date.

2. Kadimastem and iTolerance Work to Bring Immunosuppression-Free Diabetes Cell Therapy to Trials

Kadimastem and iTolerance have requested an INTERACT meeting with the FDA to discuss their preclinical joint cell therapy in development for the potential treatment of diabetes, iTOL-102.

3. Atara’s Cell Therapies Rise for EBV+ PTLD, Sink for Multiple Sclerosis After Data Updates

Atara Biotherapeutics is advancing tabelecleucel (tab-cel; Atara Biotherapeutics), its allogeneic Epstein–Barr virus (EBV)-specific T-cell immunotherapy for patients with relapsed or refractory (r/r) or treatment-naïve Epstein-Barr virus-positive post-transplant lymphoproliferative disease (EBV+ PTLD) following solid organ transplant or hematopoietic cell transplant towards biologics license application submission as well as an expanded indication. On the other hand, the company is planning to discontinue the phase 2 EMBOLD trial (NCT03283826) of ATA188, its other allogeneic, EBV T-cell immunotherapy clinical-stage candidate.

4. Taysha Doses First Pediatric Patient With Rett Syndrome With Gene Therapy TSHA-102

The first pediatric patient has been dosed in the phase 1/2 REVEAL Pediatric Study clinical trial (NCT06152237) evaluating Taysha Gene Therapies’ TSHA-102, an investigational adeno-associated virus (AAV) vector-based gene therapy, for the treatment of Rett syndrome.

5. Tome Biosciences to Make Use of Genevant’s Lipid Nanoparticle Delivery Tech

In a new collaboration agreement with a deal value of $114.3 million, Tome Biosciences has obtained a nonexclusive license to use Genevant Sciences’ lipid nanoparticle delivery technology along with its own programmable genomic integration technology in the development of a new gene editing approach to treating a rare monogenic liver indication that has yet to be specified.

6. Ferring’s Adstiladrin Hits Wide Availability in the US

More than a year after the FDA approval of Ferring’s nadofaragenefiradenovec (Adstiladrin) for high-risk Bacillus Calmette-Guérin-unresponsive non-muscle invasive bladder cancer, the gene therapy has been produced in sufficient product supply levels to allow for wide availability to patients in need. As a result, Ferring has put a wrap on the Early Experience Program through which some patients were previously able to access the therapy.

7. ElevateBio Teams Up With Xcell Biosciences on Cell Therapy Manufacturing

As part of a new collaboration, ElevateBio will be the first company to beta test Xcellbio’s AVATAR Foundry device, which is intended to help improve the potency and persistence of cell therapy products under the conditions found in the solid tumor microenvironment. ElevateBio will also have the use of Xcellbio’s AVATAR incubator system and its potency-measuring AVATAR Ai system.

8. Precision BioSciences Allows TG Therapeutics to Take on Development of Azer-cel for Noncancer Indications

In a newly completed deal, TG Therapeutics has obtained a license to develop Precision BioSciences’ azercabtagene zapreleucel (azer-cel), an allogeneic chimeric antigen receptor T-cell (CAR-T) product, for autoimmune disease and other noncancer indications. "After an extensive review of the CAR-T products available for development in immunology, we are excited to bring azer-cel into our portfolio as we look to expand our offerings for patients suffering from autoimmune diseases," Michael S. Weiss, the chairman and CEO of TG Therapeutics, said in a statement.