Around the Helix: Cell and Gene Therapy Company Updates – October 2, 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. Genprex Makes Moves to Bring its Gene Therapy Product Reqorsa to Mesothelioma

Genprex has formed a Mesothelioma Clinical Advisory Board with the intent of bringing quaratusugene ozeplasmid (Reqorsa), its investigational immunogene therapy intended to treat various forms of cancer, to mesothelioma.

2. Canada Approves Vertex and CRISPR Therapeutics’ Gene Therapy Casgevy for Sickle Cell Disease and TDT

Health Canada has granted marketing authorization to Vertex Pharmaceuticals' and CRISPR Therapeutics’ autologous gene-edited cell therapy exagamglogene autotemcel (exa-cel), marketed under the name Casgevy, for the treatment of sickle cell disease (SCD) in patients aged 12 years and older with recurrent vaso-occlusive crises (VOCs) and patients aged 12 years and older with transfusion-dependent beta thalassemia (TDT).

3. BMS and 2seventy bio Axe Trial for Ide-Cel in Newly Diagnosed Multiple Myeloma

Bristol Myers Squibb (BMS) and 2seventy bio have discontinued recruitment activities for the phase 3 KarMMa-9 clinical trial (NCT06045806), which was intended to evaluate idecabtagene vicleucel (ide-cel; marketed as Abecma) in combination with lenalidomide maintenance against lenalidomide maintenance alone for the treatment of newly diagnosed multiple myeloma in patients who had a suboptimal response following autologous stem cell transplant.

4. Atamyo Therapeutics Hits Milestones for Limb-Girdle Muscular Dystrophy Programs

Atamyo Therapeutics has finished recruiting patients for the dose escalation portion of its phase 1b ATA-001-FKRP clinical trial (NCT05224505) evaluating ATA-100, an investigational adeno-associated virus (AAV) vector-based gene therapy, for fukutin-related protein (FKRP) limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9); in addition, the company has submitted an investigational new drug (IND) application to the FDA for ATA-200, an investigational AAV vector-based gene therapy intended to treat γ-sarcoglycan-related LGMD Type 2C/R5 (LGMD2C/R5).

5. Hemophilia B Gene Therapy Nets National Reimbursement in Spain

Etranacogene dezaparvovec (Hemgenix; EtranaDez; CSL Behring, UniQure), an AAV serotype 5 vector-based gene therapy approved for the treatment of some patients with hemophilia B, has garnered a positive recommendation from the Spanish Interministerial Commission on the Pricing of Medicines, enabling national reimbursement for eligible patients. “Following this positive recommendation, our priority will now be to work with healthcare authorities to conclude the agreement and ensure Hemgenix can reach patients and healthcare professionals throughout the country," Maria Jose Sanchez Losada, the general manager of CSL Behring Iberia, said in a statement.

6. New Company InspiroGene Aims to Support Commercialization Efforts for Cell and Gene Therapy

InspiroGene, a new company launched by McKesson, is exclusively focused on supporting cell and gene therapy developers in bringing their products to the market. InspiroGene is offering services including third-party logistics programs, specialty distribution services, and biologics specialty pharmacy solutions.

7. Adicet Begins Recruitment Activities for Trial Evaluating Gamma Delta T-Cell Therapy in Autoimmune Disease

Adicet Bio's phase 1 clinical trial (NCT06375993) evaluating ADI-001, an investigational allogeneic chimeric antigen receptor (CAR)-engineered gamma delta T-cell therapy, for the treatment of autoimmune disease, is now enrolling patients with lupus nephritis. The company stated that the trial would also begin enrolling patients with systemic lupus erythematosus, systemic sclerosis, and antineutrophil cytoplasmic autoantibody associated vasculitis before the end of the year.

8. Modalis Garners Rare Pediatric Disease Designation for Congenital Muscular Dystrophy Epigenetic Editing Therapy

Modalis Therapeutics' MDL-101, an investigational epigenetic editing therapy intended to treat congenital muscular dystrophy type 1a (LAMA2-CMD), has received rare pediatric disease designation from the FDA. The therapy, which utilizes the company's CRISPR-based epigenome editing technology, is intended to upregulate expression LAMA1, a gene that produces a similar protein to the disease-targeted gene, LAMA2.