Around the Helix: Cell and Gene Therapy Company Updates – October 4, 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 Votes Down BrainStorm Cell Therapeutics’ ALS Cell Therapy NurOwn

An FDA Cellular, Tissue, and Gene Therapies Advisory Committee (AdComm) has voted against BrainStorm Cell Therapeutics’ debamestrocel (NurOwn), an investigational autologous mesenchymal stem cell neurotrophic factor–secreting cell therapy product intended to treat amyotrophic lateral sclerosis, ahead of its Prescription Drug User Fee Act (PDUFA) action date, which is slated for December 8, 2023.

2. Dose-Limiting AAV Toxicity Responsible for Cure Rare Disease’s DMD Trial Death

Investigations by Cure Rare Disease into the death of the first and only patient with Duchenne muscular dystrophy (DMD) treated with a CRISPR-based adeno-associated virus vector (AAV9) gene therapy delivering dCas9-VP64 transgene in a single patient trial (NCT05514249) have revealed that the patient died from acute respiratory distress syndrome caused by an innate immune reaction after high dose rAAV gene therapy.

3. Rocket’s LAD-I Gene Therapy BLA Accepted for Priority Review

The FDA has accepted and granted Priority Review to Rocket Pharmaceuticals’ biologics license application (BLA) for the gene therapy RP-L201 (marnetegragene autotemcel) intended for treatment of severe leukocyte adhesion deficiency-I (LAD-I).

4. New FDA Pilot Program Will Provide Hands-On Regulatory Guidance for Cell and Gene Therapy Trials in Rare Disease

The Support for clinical Trials Advancing Rare disease Therapeutics Pilot Program, an FDA initiative newly announced on September 29, 2023, includes provisions that may help smooth and speed up the path to approval for several clinical trials evaluating investigational cell therapies and/or gene therapies for rare disease indications.

5. News and Expert Insights in Rare Cancer Cell Therapy for Rare Cancer Day

For Rare Cancer Day, observed annually on September 30 by the patient and clinician communities, the CGTLive team selected some of our news coverage and interviews from the past few months on cell therapies in rare cancer, in order to offer a snapshot of the therapeutic progress being made in this field.

6. PTC Therapeutics Lets Go of Approximately 25% of Employees

The news comes several months after the company announced that it would suspend the development of several of its preclinical and early research gene therapy programs. PTC noted that positions at the company’s gene therapy facility in Hopewell, NJ, were among the groups mainly affected by the cuts. “I would like to acknowledge the tremendous contribution of the PTC team members affected by the reduction in workforce," Matthew B. Klein, MD, the CEO of PTC Therapeutics, said in a statement. "The workforce and operating expense reductions continue the efforts we began in May to focus resources on key R&D programs and our commercial enterprise.”

7. Resilience to Provide Manufacturing Services for BridgeBio Pharma’s Gene Therapies

As part of the newly announced agreement, BridgeBio will provide its manufacturing processes for its AAV gene therapy products, which include treatments for Canavan disease and congenital adrenal hyperplasia, to Resilience. Resilience may receive milestone payments and royalties, and will also be the main provider of commercial manufacturing for these products if they are approved.

8. Kate Therapeutics Enlists Capsida Biotherapeutics to Manufacture its Gene Therapy Products

Kate Therapeutics’s pipeline of AAV gene therapies includes treatments for muscle and heart disease, such as myotonic dystrophy type 1 and facioscapulohumeral muscular dystrophy. The financial details of the new arrangement have not been made public, though it is known that Capsida will receive financial compensation for its services.

9. Lexeo Therapeutics Makes Initial Public Offering

Lexeo filed for the initial public offering with the United States Securities and Exchange Commission (SEC) on September 29, 2023. The company, which is developing gene therapies for indications including Friedreich ataxia cardiomyopathy, arrhythmogenic cardiomyopathy, and Alzheimer disease, is aiming to garner $100 million with the move.

10. bluebird bio Modifies Agreement With its Manufacturer Lonza

bluebird bio filed with the US SEC on September 21, 2023, to make changes to its collaboration agreement with Lonza for the latter’s manufacturing of bluebird’s Zynteglo and Skysona gene therapy products. Among the listed modifications were a bolstering of manufacturing capacity and changes to the fee schedule and payment structure.

11. Roche to Take On Clinical Development of RNA-targeting Therapeutics Discovered by Ionis

Roche will pay Ionis $60 million upfront for exclusive rights to clinically develop, manufacture, and commercialize the 2 therapeutics, which are directed at Alzheimer disease and Huntington disease. Ionis will continue to be responsible for the remainder of the preclinical development of these products.

12. AbbVie Bows Out of Collaboration With Caribou Biosciences

According to a US SEC filing from September 26, 2023, AbbVie made the decision to end its agreement with Caribou, which involved collaborative preclinical research and manufacturing work on 2 of AbbVie’s CAR-T products for oncology indications, and which was last modified on February 9, 2021. Caribou noted in the filing that the termination was made in relation to AbbVie’s own strategic goals and was not a reaction to Caribou’s execution of the relevant tasks or the findings produced thus far.

13. Kriya Therapeutics and Everads Therapy Team Up to Tackle Geographic Atrophy, Other Indications

Under a new agreement, Kriya will incorporate Everads’ suprachoroidal delivery technology into its investigational gene therapies that target the retina for the treatment of ophthalmology indications. “Our collaboration with Everads aligns with our vision to implement next generation delivery technologies that can optimize AAV delivery to the eye via 1-time administration, which has the potential to substantially reduce patient burden associated with repeated intravitreal injections or subretinal surgical approaches,” Shankar Ramaswamy, MD, the cofounder and CEO of Kriya, said in a statement.