Around the Helix: Cell and Gene Therapy Company Updates – June 12, 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. Adicet Bio Seeks to Join Clinical Trial Stage for Lupus Therapies

Adicet Bio has received Fast Track Designation for its allogeneic gamma delta T-cell therapy ADI-001 for treating relapsed/refractory class III or class IV lupus nephritis, thus joining the race of clinical-stage candidates being developed for autoimmune diseases.

2. Taysha’s Rett Syndrome Gene Therapy Gets RMAT Designation

The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) Designation to Taysha Gene Therapies’ TSHA-102 gene therapy program for treating Rett syndrome.

3. TCR-T Therapy Cleared for AML Trial in Combination With AlloHSCT

The FDA has cleared BlueSphere Bio’s investigational new drug application for the company’s T-cell receptor T-cell (TCR-T) therapy BSB-1001, allowing the company to open the forthcoming phase 1/2a TCX-101 trial in patients with high risk refractory acute myeloid leukemia (AML), acute lymphocytic leukemia, and myeloid dysplastic syndrome, in conjunction with allogeneic hematopoietic stem cell transplantation (alloHSCT).

4. AOC Therapy Granted Breakthrough Designation for Myotonic Dystrophy Type 1

The FDA has granted Breakthrough Therapy Designation to Avidity Biosciences’ antibody oligonucleotide conjugate (AOC) therapy delpacibart etedesiran (del-desiran; AOC 1001) for treating myotonic dystrophy type 1.

5. Exothera to Shoulder Process Development and Manufacturing for Asgard’s In-vivo Gene Therapy Product

Asgard Therapeutics has chosen Exothera to handle process development and manufacturing services for AT-108, the former’s anticancer in-vivo gene therapy product that functions by reprogramming cancer cells, up to the phase 1/2 clinical trial stage. “Following our proof-of-concept studies supporting the lead candidate AT-108, we are very happy to reach process development phase and start chemistry, manufacturing, and contols activities,” Cristiana Pires, PharmD, PhD, the cofounder and CEO of Asgard, said in a statement.

6. Sino Biological and InDevR Join Forces on Multiplexed Assay Development

Under a new agreement, InDevR will be able to use Sino Biological’s reagents in its VaxArray Platform, with the intention of speeding up the development and launch of analytical tools for use in applications including mRNA therapy, mRNA vaccines, cell therapy, and gene therapy. “Our partnership with Sino Biological, with their extraordinary breadth and depth of offerings and commitment to quality, will drive new product development and empower our customers to design their own multiplex assays with ease,” Kathy Rowlen, PhD, the CEO of InDevR, said in a statement.

7. Opus Genetics Seeks to Bring IRD Gene Therapy Trials to UAE

In a newly established collaboration with the United Arab Emirate (UAE)’s Department of Health – Abu Dhabi (DoH), Opus Genetics will pursue preclinical and clinical development of gene therapy products for rare inherited retinal diseases (IRDs) in the UAE. Under the agreement, the company will gain access to data from the Emirati Genome Programme for this purpose. “By leveraging the Emirate’s advanced genomics expertise and data-enabled infrastructure, we seek to boost preclinical research and trials that will improve healthcare outcomes today and for generations to come,” Asma Al Mannaei, MBBS, MPH, the executive director of the Research and Innovation Centre at DoH, said in a statement. “On our journey to becoming a global hub for health and life science, Abu Dhabi and the Department of Health will continue to act as a both an accelerator and a platform for innovation.”