Around the Helix: Cell and Gene Therapy Company Updates – November 1, 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.

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1. FDA AdComm Favorable Towards Vertex and CRISPR’s Sickle Cell Gene-Edited Cell Therapy, but Recommends Postmarketing Research

An FDA Cellular, Tissue, and Gene Therapies Advisory Committee (AdComm) discussion was generally favorable towards Vertex Pharmaceuticals and CRISPR Therapeutics’ autologous gene-edited cell therapy exagamglogene autotemcel, an investigational treatment for sickle cell disease and transfusion-dependent β-thalassemia.

2. ELEVIDYS Study Fails Primary End Point in DMD, Secondary End Points Show Statistically Significant Benefit

Sarepta Therapeutics’ global, pivotal, phase 3 EMBARK study (Study SRP-9001-301; NCT05096221) of delandistrogene moxeparvovec-rokl gene therapy, marketed as ELEVIDYS, in children with Duchenne muscular dystrophy (DMD) has failed its primary end point according to new topline data.

3. Mustang Bio’s CAR/Oncolytic Virus Combo Cleared for Glioblastoma/Astrocytoma Trial

The FDA has accepted Mustang Bio’s investigational new drug (IND) application for a phase 1 clinical trial evaluating the combination of chimeric antigen receptor (CAR) T-cell therapy MB-101 and oncolytic virus MB-108 in patients with recurrent glioblastoma and high-grade astrocytoma, termed MB-109.

4. Ahmad Masri, MD, MS, on the Potential of Gene Therapy in ATTR Amyloidosis

For World Amyloidosis Day, observed annually on October 26 by the patient and clinician communities, CGTLive interviewed Ahmad Masri, MD, MS, a cardiologist at the Center for Hypertrophic Cardiomyopathy at Oregon Health & Science University, about the current landscape of care for transthyretin (ATTR) amyloidosis and how NTLA-2001 could potentially transform this landscape.

5. Brett Kopelan, MA, and Heather Fullmer on the Potential Impact of Gene Therapy on Epidermolysis Bullosa’s Treatment Landscape

To kick off EB Awareness Week, observed annually from October 25 to 31 by the patient and clinician community, CGTLive interviewed Brett Kopelan, MA, the executive director of the Dystrophic Epidermolysis Bullosa Research Association (debra) of America, and Heather Fullmer, the community engagement director of debra of America, about the current state of care in EB and how the advent of gene therapy could potentially change the treatment landscape for this patient population.

6. Royalty Pharma Scoops Up Royalties On SMN2 Splicing Modifier As Part of New Agreement With PTC

Royalty Pharma has purchased royalties on Roche’s Evrysdi, an orally administered survival motor neuron-2 (SMN2) splicing modifier for spinal muscular atrophy, from PTC Therapeutics. In addition to the initial purchase, which covered a portion of PTC’s royalties, the agreement includes the option for Royalty Pharma to buy half of the remaining royalties in PTC’s possession after this transaction; PTC may also sell all of its remaining royalties.

7. Cellectis and AstraZeneca Team Up to Tackle Cell and Gene Therapy Development for Various Indications

In a new collaboration, AstraZeneca is licensing Cellectis’ gene editing technologies and manufacturing ability for the purpose of developing advanced therapeutics aimed at 25 genetic targets, which may cover indications in oncology, immunology, and rare disease. From this work, up to 10 product candidates may be selected for further development, with AstraZeneca having the right to obtain an exclusive global license on the products prior to the submission of IND applications.

8. MHRA Clears CDMO ViroCell to Manufacture Viral Vectors at Great Ormond Street Hospital

The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has given contract development and manufacturing organization (CDMO) ViroCell the go ahead to produce lentivirus and gamma-retro viral vectors from within Great Ormond Street Hospital’s Zayed Centre for Research. ViroCell stated that it expects the move to help ease industry shortages for these vectors slowing down development of new cell and gene therapies.

9. Sanofi Puts Money Into Gene Therapy Developer MeiraGTx

In exchange for a purchase of MeiraGTx’s shares totaling $30 million, Sanofi has gained the right of first negotiation for the use of several of MeiraGTx’s technologies and programs. “We view Sanofi’s interest in our Riboswitch gene regulation technology and our clinical Xerostomia program as further validation of the broad potential of our vertically integrated platform and we are excited to work with Sanofi as we advance these programs,” Alexandria Forbes, PhD, the president and chief executive officer of MeiraGTx, said in a statement.

10. bluebird bio Makes Arrangements for Potential Sale of Priority Review Voucher to Undisclosed Buyer

Pending the approval of lovotibeglogene autotemcel (lovo-cel), bluebird’s gene-edited cell therapy for which the company submitted a biologics license application to the FDA with a request for priority review, bluebird may receive a vendible priority review voucher from the agency. “The potential sale of a priority review voucher would provide an important source of nondilutive capital for bluebird ahead of the anticipated launch of lovo-cel,” Chris Krawtschuk, the chief financial officer of bluebird bio, said in a statement.

11. Eli Lilly and Company Snags Rights in Beam Therapeutics and Verve Therapeutics Collaboration

Lilly has picked up codevelopment and cocommercialization rights for several of Verve’s cardiovascular disease base editing programs via a preexisting agreement between Verve and Beam. “This deal provides meaningful upfront capital to advance our portfolio of clinical- and research-stage programs, with significant additional value achievable as the Verve programs advance through development,” John Evans, MBA, the chief executive officer of Beam, said in a statement. “In parallel, it provides Verve with a world-class partner for the long term, given Lilly’s deep expertise and resources in the cardiovascular space.”