Around the Helix: Cell and Gene Therapy Company Updates – July 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. CBER To Conduct Interactive CGT Site Tours to Improve Communication, Share Best Practices

The FDA has announced a Cellular and Gene Therapies Interactive Site Tours Program (the Interactive Site Tours Program) to improve communication and share best practices between industry and the regulatory agency. The program will see regulatory managers from the Center for Biologics Evaluation and Research (CBER) touring biotechnology manufacturing facilities developing cellular and gene therapy products.

2. BrainStorm’s New Trial May Support ALS Cell Therapy BLA

BrainStorm Cell Therapeutics has reached alignment with the FDA on chemistry, manufacturing, and control aspects of a phase 3b clinical trial evaluating debemestrocel (Nurown) cell therapy for the potential treatment of amyotrophic lateral sclerosis (ALS). The news comes a couple of months after the company announced that it had reached an accord with the FDA on a phase 3b trial design for debemestrocel, the endpoints of which would support a biologics license application (BLA) approval for the therapy.

3. First Patient in US Trial Receives Nexcella’s Light Chain Amyloidosis CAR-T NXC-201

Immix Biopharma has dosed the first patient in the phase 1b/2 NEXICART-2 clinical trial (NCT06097832), the first trial in the United States evaluating subsidiary Nexcella's NXC-201 (formerly referred to as HBI0101), an investigational autologous chimeric antigen receptor T-cell (CAR-T) therapy that targets B-cell maturation antigen, for the treatment of relapsed/refractory (r/r) light chain amyloidosis.

4. Second-Generation NK Cell Engager Heading to Trial for CD33+ Leukemia

The FDA has cleared GT Biopharma’s investigational new drug application for GTB-5550, a natural killer (NK) cell engager therapy for the potential treatment of with r/r CD33 expressing hematologic malignancies, including acute myeloid leukemia and high-risk myelodysplastic syndrome.

5. Nona Biosciences and Alaya.bio Team Up to Tackle In Situ CAR-T Development

The 2 companies have agreed to collaborate on the development of CAR-T therapies that are produced by reprogramming T-cells in situ. Nona will provide the use of antibodies discovered with its HCAb Harbour Mice platform along with its site-specific conjugation technology and Alaya.bio will bring its polymeric in situ delivery platform to the table. “The quality of the targeting agents that we graft onto our polymeric nanoparticle ensures the efficacy and safety of our technology," Renaud Vaillant, MS, the CEO and cofounder of Alaya.bio, said in a statement. “With Nona Biosciences, we have identified promising new-generation candidates that clearly differentiate from what exists and from what has been used by others.”

6. Fertility Preservation Coverage for SCD Gene Therapy the Focus of Vertex’s Lawsuit Against the US Government

Vertex Pharmaceuticals, which codeveloped FDA-approved sickle cell disease (SCD) gene therapy exagamglogene autotemcel (exa-cel; marketed as Casgevy) with CRISPR Therapeutics, is suing the US government over a federal policy preventing the company from assisting patients covered by Medicaid to access fertility preservation services. The matter is of significant interest as the lymphodepleting conditioning regimen that patients must undergo before receiving the potentially curative gene therapy is known to pose a substantial risk of causing infertility.

7. Orum Therapeutics Lends the Use of its TPD² Technology to Vertex

Under a new license and option agreement, Vertex Pharmaceuticals will utilize Orum Therapeutics’ Dual-Precision Targeted Protein Degradation (TPD²) technology in discovery activities related to the development of degrader-antibody conjugates, which may be utilized for targeted conditioning in conjunction with gene editing products. As per the agreement, Vertex may choose to select up to 3 candidates it discovers with the technology for development through commercialization, in exchange for option, milestone, and royalty payments to Orum.

8. Caribou Discards NK Cell Therapy Development Plans and Lets Go of Some Staff

According to a July 16, 2024 US Securities and Exchange Commission (SEC) Filing, Caribou Biosciences has made the decision to discontinue development of its allogeneic CAR-NK cell platform, which had been in the preclinical stage. Alongside the move, the company also noted it would be laying off 21 employees, constituting about 12% of its staff. "The Company is undertaking this reduction to extend its cash runway and focus resources on its allogeneic CAR T-cell therapy platform and on rapidly advancing 4 oncology and autoimmune disease clinical programs through multiple milestones expected in 2024 and 2025," Caribou wrote in the SEC filing.