Around the Helix: Cell and Gene Therapy Company Updates – June 14, 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 Clinical Data Presented at EHA’s 2023 Congress

Catch up on CGTLive’s coverage of the European Hematology Association (EHA) 2023 Congress, held June 8-11, both virtually and in Frankfurt, Germany. Our coverage will continue throughout the week.

2. FDA Accepts Exa-cel BLA for Sickle Cell and Transfusion-Dependent β-Thalassemia

The FDA has accepted Vertex Pharmaceuticals’ biologics license application (BLA) for exagamglogene autotemcel (exa-cel) for treating severe sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). The FDA has granted Priority Review for SCD with a Prescription Drug User Fee Act (PDUFA) target action date of December 8, 2023, and Standard Review for TDT with a PDUFA target action date of March 30, 2024.

3. Patient Death Prompts Pause in 2seventy bio's Trial for Acute Myeloid Leukemia CAR-T

Seattle Children’s has paused the phase 1 PLAT-08 clinical trial (NCT05105152) evaluating 2seventy bio’s SC-DARIC33, an investigational CD33-directed chimeric antigen receptor T-cell (CAR-T) therapy intended to treat acute myeloid leukemia, following the death of the first patient treated at the study’s second dose level (5x10⁶ SC-DARIC33 T-cells/kg). Seattle Children’s and 2seventy bio, which are collaborating on the development of SC-DARIC33, are currently investigating the cause of the grade 5 serious adverse event which lead to the patient’s death and the potential for its relation to SC-DARIC33.

4. BrainStorm Cell Therapeutics Gets AdComm and PDUFA Dates for ALS Cell Therapy NurOwn

The FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee will review the supporting data of the BLA for BrainStorm Cell Therapeutics’ NurOwn, an investigational autologous mesenchymal stem cell neurotrophic factor–secreting cell therapy product intended to treat amyotrophic lateral sclerosis (ALS), in an advisory committee (AdComm) meeting on September 27, 2023, according to an announcement. In addition, the PDUFA action date for the BLA has been set by the agency for December 8, 2023.

5. Legend Biotech Seeks Expanded Use for Cilta-cel in Lenalidoimide-Refractory Multiple Myeloma

Legend Biotech has submitted a supplemental BLA for ciltacabtagene ciloleucel (cilta-cel) to include the treatment of adult patients with relapsed and lenalidomide-refractory multiple myeloma who have received at least 1 prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent.

6. Expert Insights on Gene Therapy for International Batten Awareness Day

For International Batten Awareness Day, observed annually on June 9, CGTLive reached out to experts to inquire about the current treatment landscape for Batten disease and how gene therapy could potentially impact care for this rare disease. Featured were insights from Ineka Whiteman, PhD, head of research and medical affairs, Batten Disease Support and Research Association (BDSRA) Australia, consultant to BDSRA Foundation (USA) and Beyond Batten Disease foundation, and Paulo Falabella, MD, PhD, vice president, clinical development & operations, patient advocacy, rare diseases, REGENXBIO.

7. StrataStem to Provide Donor Samples to Axol Bioscience for Alzheimer Disease Research

Axol Bioscience will utilize the patient samples, which were obtained from consenting patients with sporadic Alzheimer disease (sAD) and healthy donors and include fibroblasts and blood, to produce induced pluripotent stem cells for use in a “clinical trial in a dish” approach to evaluating treatments for sAD.

8. AAVantgarde Bio Raises €61 Million to Progress AAV Gene Therapy Programs

The funds, which were obtained from a recently closed round of Series A financing, will support the further clinical development of an adeno-associated virus (AAV)-based treatment for Usher Syndrome Type 1 B associated retinitis pigmentosa and the initiation of clinical development foran AAV-based treatment for Stargardt disease.

9. Astellas Pharma and Kate Therapeutics Join Forces to Pursue Further Development of XLMTM Gene Therapy

KT430, an investigational AAV-based gene therapy intended to treat X-linked myotubular myopathy (XLMTM) by delivering the MTM1 gene, is currently in the preclinical stage of development. “Our company uses novel technology platforms that directly address the key limitations of current gene therapies, including tissue-specific delivery and gene regulation, with the potential to improve efficacy and safety,” Kevin Forrest, PhD, the president and CEO of KateTx, said in a statement.

10. New Gene Therapy Company Beacon Therapeutics Launches With Focus on Tackling Retinal Diseases

Beacon Therapeutics, which launched with $120 million in funding provided by Syncona Limited and other investors, has acquired assets from a program entitled AGTC which originated at the University of Oxford. The lead asset obtained by Beacon is AGTC-501, an investigational gene therapy intended to treat X-linked retinitis pigmentosa, which is currently being evaluated in multiple clinical trials.

11. AstraZeneca Obtains Treg Cell Therapy Focused Exclusive Option and License Agreement With Quell Therapeutics 

The agreement will allow for AstraZeneca to carry out further development and commercialization of engineered T-regulator (Treg) cell therapy candidates that Quell is pursuing the initial development of for type 1 diabetes and inflammatory bowel disease. “This collaboration builds on our pioneering work to develop exquisitely engineered, multimodular Treg cell therapies for immune disorders and provides excellent validation for the technologies and capabilities we have established,” Iain McGill, the CEO of Quell Therapeutics, said in a statement.

12. StemCyte to Provide Raw Materials for Allogeneic CAR-NK Cell Therapy Development

StemCyte announced that it has entered a cooperation agreement with an unnamed cell therapy company based in the United States to provide umbilical cord blood for use in CAR-natural killer (NK) cell therapy. “...Being able to reliably source starting substances for cell supplies is the key for the development of cell therapy products,” Tong-Young Lee, PhD, the CEO of StemCyte, said in a statement.

13. Turnstone Biologics Pursues Initial Public Offering

Turnstone Biologics has filed with the United States Securities and Exchange Commission for an initial public offering. The company is developing tumor infiltrating lymphocyte therapies intended to treat solid tumor indications. One of Turnstone’s investigational products, TIDAL-01, is currently being evaluated in 2 phase 1 clinical trials for indications including breast cancer, colorectal cancer, and melanomas.