Around the Helix: Cell and Gene Therapy Company Updates – June 21, 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. MDA, CureDuchenne, PPMD Collaborate to Reduce AAV Antibodies to Enable Gene Therapy

CureDuchenne, Muscular Dystrophy Association (MDA), and Parent Project Muscular Dystrophy (PPMD) have just announced their collaboration on a clinical trial grant to investigate redosing of adeno-associated virus (AAV) vector-based gene therapies in patients with Duchenne muscular dystrophy (DMD). Each organization is granting $100,000 for the 1-year clinical trial at University of Florida (UF) to be led by Barry Byrne, MD, PhD, chief medical advisor, MDA, and associate chair, pediatrics and director, Powell Gene Therapy Center, UF.

2. Patient Death Prompts Hold on Arcellx’s CART-ddBCMA for R/R Multiple Myeloma

The FDA has placed a clinical hold on Arcellx’s investigational new drug application for CART-ddBCMA after a patient with relapsed/refractory (r/r) multiple myeloma treated in the phase 2 iMMagine-1 clinical trial (NCT05396885) died. The FDA has cleared Arcellx to continue dosing patients that have undergone lymphodepletion.

3. BioSenic Puts Trial for MSC-Derived Therapy ALLOB on Hold Following Disappointing Efficacy Results

BioSenic has paused its phase 2b trial clinical trial (NCT04432389) evaluating ALLOB, an investigational allogeneic mesenchymal stem cell (MSC)-derived therapy intended to accelerate healing of bone fractures, in response to negative results obtained for the study’s primary efficacy end point.

4. 2023 World Sickle Cell Day: The Home Stretch for Gene Therapy in SCD?

On this year’s World Sickle Cell Day, gene and cell therapies for treating sickle cell disease (SCD) are closer to the market than ever. CGTLive took a look at the novel therapies closest to market for the disease and their progress and challenges, with insights from companies developing these therapies and clinicians.

5. ProKidney Adds New Manufacturing Facility to Support Development of its Chronic Kidney Disease Cell Therapy

The 210,000 square foot Greensboro North Carolina facility will provide support for the further development and potential commercialization of REACT, an autologous cell therapy currently being evaluated in a phase 3 clinical trial for the treatment of patients with chronic kidney disease. The purchase, which also includes approximately 22 acres of land, will cost the company approximately $25.5 million.

6. NIH’s NHLBI CATALYZE Program Approves Additional Funding for Ambulero’s Vascular Disease Gene Therapy Research

This second phase of funding provided by the National Institutes of Health (NIH)’s NHLBI CATALYZE program will enable research being carried out by Omaida C. Velazquez, MD, FACS, DFSVS, the chief medical officer of Ambulero, and Zhao-Jun Liu, MD, PhD, the chief scientific officer of Ambulero. The research is expected to support further development of the company’s gene and cell therapy platform for vascular diseases.

7. FDA Clears Ferring Pharmaceuticals to Scale Up Its Manufacturing of ADSTILADRIN

The FDA has approved a Prior-Approval Supplement to the Biologics License Application for ADSTILADRIN, Ferring Pharmaceuticals’ gene therapy for non-muscle invasive bladder cancer. The company expects that ADSTILADRIN, which received an FDA approval in December 2022, will reach initial commercial availability in the United States in the second half of 2023. 

8. Verve Therapeutics Joins Forces With Eli Lilly and Company on Atherosclerotic Cardiovascular Disease Gene Editing Program

Under the terms of a new research collaboration agreement, Verve Therapeutics will be responsible forcarrying out advancement of its lipoprotein (a)-targeted in vivogene editing program, which is currently in preclinical development, through phase 1 clinical development. Any subsequent research, manufacturing, and commercialization activities will be handled by Eli Lilly.

9. uniQure and Partner CSL Make Milestone Commercial Sale of Hemgenix

Hemgenix (etranacogene dezaparvovec) is an AAV vector-based gene therapy intended to treat hemophilia B which is currently approved for use in the United States and several other jurisdictions. “The first commercial sale in the US is a major milestone for uniQure as it marks the fulfillment of our promise to deliver genetic medicines that have the potential to transform people’s lives,” Matt Kapusta, the chief executive officer of uniQure, said in a statement.

10. REGENXBIO and University of Pennsylvania File Patent Infringement Complaint Related to Sarepta Therapeutics’ SRP-9001

The complaint relates to theuse of AAV gene therapy technology developed by the University of Pennsylvania. "We believe that Sarepta unlawfully exploited groundbreaking inventions and has continued to use them without a license," Patrick Christmas, the chief legal officer of REGENXBIO, said in a statement. SRP-9001, which is nearing the commercialization stage for treatment of DMD, has a June 22, 2023, PDUFA date.

11. Codiak Biosciences Sells AAV Technology Platform to Evox Therpaeutics

The deal for the engEx-AAV technology platform also provides Evox Therapeutics with intellectual property and certain other rights. “This is part of our long-term business strategy of leveraging improved exosome-mediated delivery to create and enable genetic medicines such as gene therapy and genome editors,” Per Lundin, PhD, the chief business officer and co-founder of Evox Therapeutics said in a statement.

12. FDA Approves Sarepta's Landmark DMD Gene Therapy Elevidys

The FDA has approved Sarepta Therapeutics’ delandistrogene moxeparvovec (SRP-9001) for treating ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) and a confirmed mutation in the DMD gene, excluding patients with any deletion in exon 8 and/or exon 9, to be marketed under the name Elevidys. "It is gratifying and a relief to finally have an approved gene therapy for DMD. This drug is likely to be the most potent treatment method available today, although it is not as robust as many had hoped. Nonetheless, approval sets the stage for intensive analysis of the long-term effects of this gene therapy, and will hopefully spur increased research and testing of improved next generation gene therapies," Jeffrey Chamberlain, PhD, professor and director, Sen. Paul D. Wellstone Muscular Dystrophy Specialized Research Center, University of Washington School of Medicine, told CGTLive.