Around the Helix: Cell and Gene Therapy Company Updates – January 3, 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. Oncternal Therapeutics Proposes Protocol Changes Following Death of Patient Treated With CAR-T Therapy ONCT-808 in Phase 1/2 Trial

An 80-year-old patient has died following treatment with Oncternal Therapeutics’ ONCT-808, an investigational chimeric antigen receptor T-cell (CAR-T) therapy being evaluated for the treatment of relapsed/refractory aggressive B-cell lymphoma in the phase 1/2 ONCT-808-101 clinical trial (NCT05588440). The company has announced that it is seeking to modify study protocols in response to the patient’s death.

2. CGTLive's Clinical Trials to Watch in 2024

On January 1, 2024, CGTLive highlighted just some of the trials we're looking forward to seeing data from in the coming year.

3. Phase 3 Trial of Gene Modifier Therapy for Retinitis Pigmentosa Ready For 2024

Ocugen has aligned with the FDA on key aspects of its proposed phase 3 trial design to further assess its gene modifier therapy, OCU400, in patients with retinitis pigmentosa caused by RHO (rhodopsin) and other gene mutations.

4. Alliance for Cancer Gene Therapy 's Highlights From the International Oncolytic Virotherapy Conference

Take a look back at the 2023 conference, hosted by the University of Calgary and McMaster University, and held in Banff, Alberta, Canada.

5. BioMarin Makes Moves to Strategize for the Future

BioMarin, whose gene therapy for Hemophilia A became FDA-approved last year, is taking several steps to strategize for the future. Among these are additions to its board of independent directors, forming a new Strategic and Operating Review Committee, and joining a Cooperation Agreement with Elliott Investment Management L.P.

6. Sanofi Excercises Additional Option in Collaboration With Scribe Therapeutics

Under a preexisting research collaboration with Scribe Therapeutics, which was originally established in 2022 and expanded in 2023 to focus on the development of in vivo CRISPR-based therapeutics, Sanofi has exercised its option for an undisclosed second target. The first target, announced in 2023, is sickle cell disease.

7. JW Therapeutics and 2seventy bio Throw Their Hat Into the Ring for CAR-T for Autoimmune Disease

JW Therapeutics and 2seventy bio have announced an expansion of their previously existing collaboration agreement that will focus on the codevelopment and commercialization of a CAR-T therapy product intended to treat B-cell driven autoimmune diseases. Under the new agreement, JW will first focus on initial process development and conducting a first-in-human clinical trial in China, and may be eligible to receive milestone payments from 2seventy.

8. Lion TCR to Leverage MaxCyte's Technologies for TCR T-cell Development

Under a new agreement, Singapore-based company Lion TCR has obtained a nonexclusive license for the utilization of MaxCyte’s Flow Electroporation technology and ExPERT platform in its efforts to develop T-cell receptor (TCR) T-cell therapies for the treatment of cancers including hepatocellular carcinoma, as well as for viral infections. "Through this important partnership, we will be able to help provide the opportunity for patients in Asia (and ultimately patients worldwide) to benefit from these innovative mRNA-based TCR T-cell treatments and continue supporting the development of new therapies for patients with solid tumors," Maher Masoud, JD, BS, the president and CEO of MaxCyte, said in a statement.