Around the Helix: Cell and Gene Therapy Company Updates – August 30, 2023

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Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

CGTLive Around the Helix

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 Bristol Myers Squibb’s Reblozyl for Expanded Indication in Myelodysplastic Syndromes

The FDA has approved Bristol Myers Squibb’s first-in-class erythroid maturation agent luspatercept-aamt (Reblozyl) as a first-line treatment for anemia in adult patients with very low, low-, or intermediate-risk myelodysplastic syndromes who may require red blood cell transfusions.

2. Poseida's Dual-Targeted Allogeneic CAR T Trial Cleared for B-Cell Malignancies

The FDA has cleared Poseida Therapeutics’ investigational new drug application for P-CD19CD20-ALLO1, its first allogeneic dual chimeric antigen receptor (CAR)-T cell therapy candidate, which targets both CD19 and CD20 antigens for the treatment of relapsed or refractory B-cell malignancies.

3. TSHA-102 Gets FDA Fast Track Designation in Rett Syndrome

The FDA has given a fast track designation to Taysha Gene Therapies’ adeno-associated virus (AAV) vector gene transfer therapy, TSHA-102, for the treatment of Rett syndrome. The agent is currently being assessed in the Canadian-based phase 1/2 REVEAL trial (NCT05606614) in adult patients with Rett syndrome.

4. Allogeneic Natural Killer Cell Trial in Solid Tumors Doses First Patient

The first patient has been dosed in a phase 1 clinical trial (NCT05990920) evaluating NKGen Biotech’s SNK02, an allogeneic natural killer (NK) cell therapy intended to treat solid tumors. SNK02, which is comprised of NK cells isolated from the peripheral blood mononuclear cells of healthy donors that are expanded ex vivo, is manufactured using a proprietary process capable of producing hundreds of thousands of potential doses from a single donation.

5. Celularity to Provide Research Support for Regeneron

Efforts under the new research collaboration services agreement will first center on advancing the development of Regeneron’s targeted allogeneic γδ CAR-T for solid tumors. “We believe that this relationship paves the way for future industry collaborations leveraging our world class cell therapy facilities and capabilities,” Robert J. Hariri, MD, PhD, the founder, chairman, and CEO of Celularity, said in a statement.

6. Notch Therapeutics Lets Go of Employees at Vancouver Lab

Notch Therapeutics has stated plans to close its lab in Vancouver and announced its intention to tighten its focus on its development of its induced pluripotent stem cell-derived T-cells at its sites in Toronto and Seattle. The company cited “externally challenging and turbulent” economic conditions and the need to maintain operational efficiency as factors in its decisions.

7. Abata Therapeutics Expands Partnership With ElevateBio to Support Development of New Treg Cell Therapy

The newly announced regulatory T-cell (Treg) therapy, ABA-201, is currently being evaluated in preclinical research for the treatment of type 1 diabetes (T1D) in patients who have remaining β cell function. “We are rapidly developing ABA-201 for T1D and expect to begin clinical studies in 2025...” Samantha Singer, MS, MBA, the president and CEO of Abata, said in a statement. “We’re also thrilled to announce the extension of our partnership with ElevateBio. The successful partnership on our lead program affords the advantage of a ‘plug-and-play’ process that will greatly accelerate the advancement of the ABA-201 program.”

8. XNK Therapeutics Joins Forces With Karolinska University for Ex Vivo NK Cell Therapy Research

Karolinska University Hospital will assist XNK Therapeutics in carrying out nonclinical large-scale bioreactor culture research intended to assess the potential of the company’s XNK02, an investigational NK cell therapy intended to treat acute myeloid leukemia. XNK Therapeutics is also engaged in a separate, previously established preclinical research project for XNK02 with the University of Texas MD Anderson Cancer Center.

9. bit.bio Enlists the Aid of Synthego in Optimizing Engineering Platform

Synthego intends to incorporate a multiplexed genetic editing strategy into a precision cell programming technology, dubbed opti-ox, that the companies believe may cut up to 10 months off engineering and development timelines. “We are thrilled to deepen our partnership with bit.bio to facilitate a leap forward in the development of synthetic biology-based cell therapies," Paul Dabrowski, MS, the CEO of Synthego, said in a statement.

10. DB-OTO Nabs FDA Fast Track Designation

Decibel Therapeutics’ gene therapy for otoferlin-related hearing loss, DB-OTO, has previously garnered orphan drug designation (ODD) and rare pediatric disease designation from the FDA, in addition to an ODD from the European Medicines Agency. The treatment is currently being evaluated in a phase 1/2 dose escalation clinical trial (NCT05788536) that is recruiting at sites in the United States and Spain.

11. Sarepta Invests in Cardiovascular Gene Therapy Through Lexeo Therapeutics

Sarepta Therapeutics has made a strategic investment, with financial details not publically disclosed, in Lexeo Therapeutics’ AAV gene therapy programs for cardiovascular diseases. The 2 companies intend to collaborate towards further development in this space. “Our recent advancements in next-generation capsid development and suspension-based manufacturing, including MyoAAV, will give us the capabilities to develop treatments for large, under-served indications including cardiomyopathies,” Doug Ingram, JD, the president and CEO of Sarepta, said in a statement.

12. Ferring Pharmaceutical Snags $500 Million Investment in its Bladder Cancer Gene Therapy

Royalty Pharma has agreed to make an investment that will consist of a $300 million upfront payment and a potential $200 million milestone payment in Ferring Pharmaceutical’s nadofaragene firadenovec-vncg (marketed as Adstiladrin), a gene therapy indicated for adult patients with high-risk, Bacillus Calmette-Guérin-unresponsive nonmuscle invasive bladder cancer with carcinoma in situ with or without papillary tumors. In return for the investment, Royalty Pharma will receive royalties on US net sales of nadofaragene firadenovec-vncg, which will be accessible to patients via an early experience program in September 2023.

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