Around the Helix: Cell and Gene Therapy Company Updates – May 3, 2023


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.

1. New Expert Insights at WOCTC

Catch up on CGTLive’s interview coverage of the World Oncology Cell Therapy Congress (WOCTC) held April 25-26 in Boston, Massachusetts.

2. Non–Small Cell Lung Cancer Cell Therapy Receives FDA IND Clearance

Kiromic BioPharma’s KB-GDT-01 (Deltacel), an investigational allogeneic gamma delta T-cell therapy intended to treat non–small cell lung cancer, has received clearance of its investigational new drug (IND) application by the FDA.

3. Liso-cel Meets Primary End Point in Follicular and Mantle Cell Lymphoma Trials

Bristol Myers Squibb’s lisocabtagene maraleucel (liso-cel; Breyanzi), a marketed CD19-directed chimeric antigen receptor T-cell (CAR-T) therapy, has met the primary end point of overall response rate in 2 separate clinical trials for patients with relapsed/refractory (r/r) follicular lymphoma (TRANSCEND FL; NCT04245839) and r/r B-cell non-Hodgkin lymphoma (TRANSCEND NHL 001; NCT02631044), respectively.

4. X-Linked Retinoschisis Gene Therapy Cleared for First-in-Human Trial

The FDA has cleared Atsena Therapeutics’ IND for the gene therapy ATSN-201 to be evaluated in the first-in-human, phase 1/2 Lighthouse study in patients with X-linked retinoschisis.

5. Teknova Launches New Buffer Screening Kit for use in Gene Therapy Process Development

The AAV-Tek AEX Buffer Screening Kit is the first product in a newly announced line intended to streamline AAV gene therapy development. “By addressing key bioprocessing bottlenecks with new, custom products, we can collaborate closely with our customers to help accelerate breakthroughs,” Stephen Gunstream, the president and CEO of Teknova, said in a statement.

6. Eterna Therapeutics’ Picks Up Exacis Biotherapeutics’ Cell Therapy Platform and Products

The acquisition provides Eterna with exclusive global rights to produce induced pluripotent stem cell (iPSC)-derived mRNA-engineered natural killer and T-cell therapies with Exacis Biotherapeutics’ global immuno-oncology platform. Exacis Biotherapeutics’ pipeline of engineered cell therapy programs for cancers is also included in the acquisition.

7. Bristol Myers Squibb Exercises First of 4 Options From 2019 Agreement With Immatics

Bristol Myers Squibb has exercised an option to acquire rights to a T-cell Receptor engineered T-cell candidate from Immatics. The collaboration agreement, originally established in 2019 and expanded in 2022, gives Bristol Myers Squibb the right to carry out further development and commercialization of the product.

8. Forge Biologics and Labcorp Join Forces to Provide Gene Therapy Development and Manufacturing Services

“We are delighted to announce this strategic collaboration, which leverages Forge’s extensive AAV manufacturing services and Labcorp’s deep scientific and drug development expertise to provide a more integrated CRO-CDMO experience supporting the advancement of genetic medicines,” John Maslowski, the chief commercial officer of Forge Biologics, said.

9. Astellas Pharma to Subsume Retinal Disease Focused Company Iveric Bio

The acquisition deal, which is worth $5.9 billion, will put Iveric’s pipeline in the hands of Astellas Pharma. While Iveric’s most advanced candidate is a protein inhibitor, the company has early-stage AAV gene therapy programs in development for Leber congenital amaurosis type 10, autosomal recessive Stargardt disease, and Usher syndrome type 2A.

10. AstraZeneca to Investigate use of Sernova’s Cell Pouch System as a Platform for New Therapies

The collaboration will involve preclinical evaluation of the Cell Pouch System used in combination with AstraZeneca’s cell therapies for multiple indications. “By engrafting functional therapeutic cells within an implanted Cell Pouch, which naturally vascularizes with surrounding tissues, we create an environment for the production and release of absent or under-expressed hormones and proteins,” Philip Toleikis, the president and CEO of Sernova, stated.

11. MicrofluidX’s Cyto Engine the Centerpiece of New Manufacturing Process Collaboration With Immatics

The 2 companies will seek to improve efficiency in manufacturing processes for cell therapies and gene therapies. As part of the collaboration, the Cyto Engine platform will be evaluated for its ability to automate production of TCR T-cells.

12. BrightPath Biotherapeutics and Artisan Bio Team Up to Push iNKT Cell Therapies Towards Clinical Trials

BrightPath Biotherapeutics will utilize Artisan Bio’s STAR-CRISPR editing platform, which it has gained non-exclusive rights to use, in the advancement of its invariant natural killer T (iNKT) cell therapy programs for various indications. "Artisan's STAR-CRISPR system, and their leading editing efficiencies in iPSC cells, will enable BrightPath's iNKT cell therapies to solve the persistence and efficacy problems faced by first generation allogeneic CAR-T programs,” Kenichi Nagai, the CEO of BrightPath, said.

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