Around the Helix: Cell and Gene Therapy Company Updates – January 10, 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 Activity Recap: December 2023 Features 2 Major Approvals, Meetings, and Clinical Holds

Last month, December 2023, the CGTLive™ team diligently tracked the FDA's activities related to the development of cell and gene therapies for the treatment of rare, complex, and otherwise challenging diseases and disorders.

2. Vertex Pauses Diabetes Cell Therapy Trial After Unrelated Patient Deaths

Vertex Pharmaceuticals has placed its diabetes type 1 cell therapy program on a protocol-specified pause following 2 patient deaths that occurred in the phase 1/2 clinical trial (NCT04786262). Vertex has stated that the deaths were unrelated to VX-880 therapy.

3. Kyverna’s CAR-T KYV-101 Cleared for US Trial in Multiple Sclerosis, Marking Seventh IND Clearance

Kyverna Therapeutics’ KYV-101, an investigational CD19-directed chimeric antigen receptor T-cell (CAR-T) therapy, has received clearance of an investigational new drug (IND) application from the FDA for a new clinical trial in patients with multiple sclerosis.

4. Pfizer’s Hemophilia B Gene Therapy Approved as Beqvez in Canada

Health Canada has approved Pfizer Canada’s gene therapy fidanacogene elaparvovec under the name Beqvez for treating patients with moderately severe to severe hemophilia B with congenital Factor IX deficiency who are negative for neutralizing antibodies to variant adeno-associated virus (AAV) serotype Rh74.

5. Sensorion and Institut Pasteur Extend Their Hearing Loss Gene Therapy Collaboration to 2028

Sensorion and Institut Pasteur have agreed to extend their existing collaboration agreement, which is focused on preclinical development, up to December 31, 2028. The collaborators intend to pursue the development of additional gene therapies beyond the 2 programs already underway, SENS-501 (OTOF-GT) and GJB2-GT.

6. PGI-Focused Company Tome Biosciences Takes Replace Therapeutics Into the Fold

Under a new agreement with a deal value of up to $185 million, Tome Biosciences will acquire Replace Therapeutics, a biotech working on a ligase-mediated programmable genomic integration (L-PGI) method for small DNA sequences. “The addition of L-PGI to our PGI toolset provides us with the flexibility to choose the right editing tool for the right indication, expanding the types of diseases that we have the potential to cure,” Rahul Kakkar, MD, the president and CEO of Tome, said in a statement.

7. Vertex Pharmaceuticals Subsidiary ViaCyte Backs Out of Diabetes Collaboration

ViaCyte has chosen to opt-out of a collaboration with CRISPR Therapeutics in which the companies were working on gene-edited stem cell therapies for diabetes. Following the opt-out, which will go into effect in early February, CTX211 (formerly VCTX211) and any other assets resulting from the collaboration will pass fully into the hands of Vertex, with the stipulation that royalties on future sales will be owed to ViaCyte.

8. AbbVie and Umoja Biopharma Join Forces to Develop In-Situ Generated CAR-T Products

Under 2 separate option and license agreements, AbbVie may license Umoja’s existing investigational CD19-directed in-situ generated CAR-T products and the 2 companies will develop up to 4 new in-situ generated CAR-T products aimed at discovery targets of AbbVie’s choice. Umoja’s existing investigational products include UB-VV111, a CD19-directed in-situ generated CAR-T product for hematologic malignancies currently in the IND-enabling stage of preclinical research.

9. Ginkgo and Biogen Meet the Goal of their Gene Therapy Manufacturing Collaboration

Ginkgo Bioworks and Biogen originally announced their goal to enhance AAV production titers for the latter company's gene therapy manufacturing process in May 2021. "Significant improvements like these are the result of a platform-based approach to biological R&D," Narendra Maheshri, PhD, the vice president of Mammalian Engineering at Ginkgo Bioworks, said in a statement. "They are possible because our program leads work closely with our collaboration partners to take full advantage of the broad scale and deep sophistication of our highly-automated foundry and growing codebase."