Around the Helix: Cell and Gene Therapy Company Updates – May 8, 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. Taysha Gene Therapies Announces RMAT Designation for TSHA-102 in Rett Syndrome

The RMAT designation comes after the FDA’s review of available safety and efficacy data from the first three patients dosed with the low dose of TSHA-102 across both REVEAL Phase 1/2 trials among adolescent/adult and pediatric patient populations. This designation enables increased dialogue with the FDA to support the potential expedited development and review of TSHA-102 in clinical evaluation for Rett syndrome.

2. OverT Bio Raises $16 Million for Next-Generation Therapy Development

The data-driven company announced it has raised $16 million in seed funding, coled by ARTIS Ventures and Wing VC, with participation from Fusion Fund, OMX Ventures, Alexandria Venture Investments, Gaingels, Civilization Ventures, Hawktail, and Cancer Research Institute. The funding will go toward expanding discovery platforms focused on addressing the primary barriers for cell therapy to create durable and curative treatments for advanced solid tumors.

3. AstraZeneca completes equity investment agreement with Cellectis

First announced in November 2023, the equity investment and a research collaboration agreement will leverage the Cellectis gene editing technologies and manufacturing capabilities to design up to 10 novel cell and gene therapy products for areas of high unmet need, including oncology, immunology, and rare diseases. Cellectis is also eligible to receive an investigational new drug option fee and development, regulatory, and sales-related milestone payments, ranging from $70M up to $220M, per each of the 10 candidate products, plus tiered royalties.

4. Vertex Positive on Casgevy Launch in Sickle-Cell Disease

Vertex Pharmaceuticals noted in its Q1 statements that after approvals late in 2023 for exagamglogene autotemcel (Casgevy), 5 patients have had stem cells collected as of mid-April in preparation for manufacturing of the gene therapy. Stuart Arbuckle, chief operating officer, noted thay “many patients have [also] begun the treatment journey” in the markets where Casgevy is approved, which include the United States, Europe, and the Middle East.

5. Metagenomi Regain Development Rights to Wholly-Owned Base Editing and RIGS Systems

The precision genetic medicines company with a proprietary, comprehensive metagenomics-derived gene-editing toolbox, announced it has regained full global rights to research, develop, manufacture, and commercialize its wholly-owned gene editing technologies, as well as its base editors and RNA-mediated integration systems (RIGS)—previously subject to exclusive rights granted to Moderna. Metagenomi and Moderna have mutually agreed to terminate their collaboration on primary hyperoxaluria type 1 (PH1).

6. Astellas and Poseida Therapeutics Enter Into Research Collaboration and License Agreement to Develop Novel Allogeneic Cell Therapies in Oncology

Astellas Pharma and Poseida Therapeutics announced that Xyphos Biosciences (an Astellas subsidiary) and Poseida have entered into a research collaboration and license agreement to develop novel convertibleCAR programs by combining the innovative cell therapy platforms from each of the companies. Xyphos utilizes a novel and proprietary ACCELTM technology*1 platform that uses its convertibleCAR (convertible Chimeric Antigen Receptor)*1 in combination with proprietary MicAbodies*1 to target tumor cells.