Around the Helix: Cell and Gene Therapy Company Updates – June 5, 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.

Catch up on CGTLive's coverage of the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting, held May 31 to June 4, in Chicago, Illinois.

The FDA has approved Bristol Myers Squibb’s lisocabtagene maraleucel (liso-cel, marketed as Breyanzi), an autologous CD19-directed chimeric antigen receptor (CAR) T-cell therapy, for treating adults with relapsed/refractory (r/r) mantle cell lymphoma (MCL) who have been previously treated with at least 2 lines of systemic therapy, including a Bruton tyrosine kinase (BTK) inhibitor.

The fourth patient with non–small cell lung cancer (NSCLC) who was treated with Kiromic BioPharma’s KB-GDT-01 (Deltacel), an investigational allogeneic gamma delta T-cell (GDT) therapy, in the phase 1/2 Deltacel-01 clinical trial (NCT06069570) has experienced disease stabilization and tumor size reduction.

Ultragenyx’s DTX401, an investigational adeno-associated virus serotype 8 (AAV8) vector gene therapy expressing the human G6PC gene, significantly reduced daily cornstarch intake compared to placebo treatment in patients with glycogen storage disease type Ia (GSDIa) who were treated in the phase 3 GlucoGene clinical trial (NCT05139316).

Anocca AB, a company focused on the development of T-cell receptor-engineered T-cell (TCR-T) therapies, and Shinobi Therapeutics, which is focused on the development of induced pluripotent stem cell (iPSC)-derived CD8 αβT-cell therapies (iPS-T), are seeking to combine their respective technologies in pursuit of allogeneic TCR engineered iPS-T-cell therapies (TCR-iPS-T). The companies intend to apply TCR-iPS-T, which will constitute a new class of T-cell therapy, to the treatment of solid tumor indications.

The nonprofit organization Caring Cross and Indian cell and gene therapy company ImmunoAdoptive Cell Therapy (ImmunoACT) have agreed to collaborate on the development and potential commercialization of TriCAR-T, a CAR-T product that targets multiple antigens and is intended for use in patients whose disease has relapsed after treatment with CD19-targeted CAR-T therapy for leukemia or lymphoma. ImmunoACT will leverage its own infrastructure in India to handle clinical development, manufacturing, and commercialization activities for TriCAR-T.

NewBiologix has launched NBX-HEK293, a proprietary cell line intended for use by its customers in the production of recombinant AAVs for gene therapy products. NBX-HEK293 has been completely sequenced and annotated and was designed for optimized doubling time and speedy recovery following single-cell cloning.

Referred to as the "Applied Gene and Cell Therapy Center", Cincinnati Children's has plans to build the new facility via renovation of an 111,000 square foot building that previously served as an auto parts distribution facility in Sharonville, Ohio. Work on the project is slated to begin in June 2024 and has an expected timeframe for completion in Summer 2025. The facility is ultimately expected to create approximately 24 new employment positions.