Around the Helix: Cell and Gene Therapy Company Updates – March 29, 2023

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 Data Presented at the 2023 MDA Clinical & Scientific Conference

Catch up on CGTLive's coverage of the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, held in Dallas, Texas, March 19-22, 2023.

2. FDA Revisits Need for Advisory Committee on Sarepta’s DMD Gene Therapy

In advance of the upcoming regulatory action deadline for Sarepta Therapuetics’ investigational gene therapy for the treatment of Duchenne muscular dystrophy (DMD), delandistrogene moxeparvovec (also known as SRP-9001), the FDA has determined it will hold an advisory committee (AdComm) meeting.

3. Janssen No Longer Seeking Approval for Carvykti in the UK

Janssen is no longer seeking the approval of ciltacabtagene autoleucel (cilta-cel; Carvykti), a chimeric antigen receptor T-cell (CAR-T) therapy for the treatment of relapsed/refractory (r/r) multiple myeloma (MM), by the UK’s National Institute of Health and Care Excellence, according to the charity group Myeloma UK.

4. Death Prompts CAR-NKT Cell Therapy Hold On Neuroblastoma Trial

The FDA has placed Athenex’s autologous GD2 chimeric antigen receptor natural killer T-cell (CAR-NKT) therapy KUR-501 on clinical hold after reports of a patient death in the phase 1 GINAKTI2 study (NCT03294954) assessing the therapy in relapsed/refractory high-risk neuroblastoma

5. FDA Finally to Discuss NurOwn MSC-NTF Cell Therapy for ALS

The FDA will hold an advisory committee meeting discussing BrainStorm Cell Therapeutics’ mesenchymal stem cell neurotrophic factor (MSC-NTF) cell therapy NurOwn for the potential treatment of amyotrophic lateral sclerosis (ALS), the company has announced.

6. Teknova, Sartorius BIA Separations to Collaborate on Streamlining Downstream Purification Process for Gene Therapy Development

The collaboration is intended to provide companies developing gene therapies with necessary consumables and a protocol for the downstream processing workflow.

7. Orgenesis, UC Davis to Collaborate on Bringing Orgenesis Mobile Processing Units and Labs to California Universities

The Orgenesis Mobile Processing Units and Labs are intended to allow for more cost-effective development of cell and gene therapies at universities and other institutions.

8. Abeona Therapeutics Announces Preclinical Development of Gene Therapies for Stargardt Disease, X-linked Retinoschisis, and Autosomal Dominant Optic Atrophy

The 3 gene therapies will utilize adeno-associated virus (AAV)-capsid based delivery.

9. MDA Invests in Gene Therapy Safety Research

The organization has provided 2 grants with a combined value of approximately $400,000 for the purpose of investigating transgene-triggered safety concerns in gene therapy.

10. Thermo Fisher Scientific to Provide Manufacturing Process Support for Arsenal Biosciences

Arsenal Bioscience is developing autologous T-cell therapies for indications including platinum-resistant ovarian cancer.

11. New Gene Therapy Company Inspire Biotherapeutics Launched

The newly formed company will focus initial efforts on developing AAV vector-based therapies for monogenic and acquired lung diseases.

12. Takeda to Utilize Artisan Bio's Genome Editing Tools in the Development of Allogeneic Cell Therapies

The agreement builds upon a previously established collaboration between the 2 companies.

13. Sonoma Biotherapeutics, Regeneron Join Forces to Develop Regulatory T-cell Therapies

The 2 companies will focus their efforts on discovery and development of new treatments for autoimmune diseases.

14. Vertex Therapeutics Enters Into Agreement With CRISPR Therapeutics for Use of CRISPR/Cas9 Gene Editing Technology in Cell Therapies

Vertex Therapeutics is developing hypoimmune cell therapy products for the treatment of type 1 diabetes.

15. New Company Seamless Therapeutics Launches With Focus on Gene Editing Treatments for Severe Diseases

The company, which received $12.5 million in a recent seed financing round, will seek to develop treatments based on the reprogramming of recombinases.

16. Canon Acquires Cell Product Manufacturing Technology From Kyoto Seisakusho

Canon has announced its intention to expand its Medical Group's activities into the regenerative medicine space.

17. Non-viral Genetic Medicines the Focus of New Collaboration Between Moderna and Generation Bio

The agreement includes options for immune cell and liver programs.

18. National Science Foundation's Engineering Research Center for Cell Manufacturing Technologies Recruits Mission Bio

Mission Bio will provide use of its Tapestri Platform for cell therapy development processes.

19. Chimeric Therapeutics Announces Positive Feedback From FDA for Phase 1 Cell Therapy Trial

CHM 2101 is an investigational CAR-T therapy intended to treat gastrointestinal and neuroendocrine cancers. Chimeric had a positive pre-IND meeting with the FDA relating to its proposed phase 1 clinical trial.

20. BetaLife Acquires iPS Cell Technology from the Agency for Science, Technology and Research

The 2 organizations will utilize the technology in the collaborative development of a new diabetes treatment.