Around the Helix: Cell and Gene Therapy Company Updates – March 27, 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. FDA Deems Mesoblast’s Data on Remestemcel-L Cell Therapy Sufficient for BLA Submission in Pediatric SR-aGVHD

The FDA has informed Mesoblast that the available data from the phase 3 MSB-GVHD001 clinical trial (NCT02336230) is sufficient for the submission of a biologics license application (BLA) for its allogeneic mesenchymal stromal cell therapy, remestemcel-L, for the treatment of children with steroid-refractory acute graft versus host disease (SR-aGVHD).

2. SRP-9001 Improves DMD Disease Trajectory, But Falls Short on Primary End Point

Data presented at the 2024 Muscular Dystrophy Association Clinical and Scientific Conference, held March 3-6, in Orlando, Florida, showed that Sarepta Therapeutics’ gene therapy delandistrogene moxeparvovec (Elevidys; previously known as SRP-9001) failed to meet the primary end point in part 1 of the phase 3 EMBARK study (NCT05096221) in Duchenne muscular dystrophy (DMD). Although, the data suggested positives in secondary analyses such as time to rise, microdystrophin expression, and 10-meter walk/run.

3. Synthekine Cleared to Tackle Lupus With CAR-T and IL-2 Combination Therapy in US Clinical Trial

Synthekine’s SYNCAR-001 + STK-009, a combination therapy consisting of an autologous CD19-directed chimeric antigen receptor T-cell (CAR-T) product that expresses an engineered IL-2 receptor (SYNCAR-001) and an engineered pegylated IL-2 cytokine (STK-009), has received clearance of an investigational new drug application from the FDA for clinical evaluation in patients with nonrenal systemic lupus erythematosus and lupus nephritis.

4. BlueRock Therapeutics' Parkinson Disease Cell Therapy Bemdaneprocel Shows Safety and Efficacy at 18 Months Posttreatment

BlueRock Therapeutics’ bemdaneprocel (BRT-DA01), an investigational neuronal cell therapy intended to treat Parkinson disease, has continued to show safety and promising initial results in terms of efficacy at 18 months posttreatment in patients who received the therapy in an ongoing phase 1 clinical trial (NCT04802733).

5. Fiocruz and Caring Cross Join Forces to Increase Accessibility of CAR-T in Brazil

As part of the agreement, nonprofit Caring Cross and Fiocruz, a Brazilian government foundation, will work together to establish local manufacturing of CAR-T products for leukemia and lymphoma. The agreement also includes a focus on advancing the development of an investigational CAR-T product for HIV infection.

6. Polyplus Launches Off-the-Shelf Plasmid pPLUS AAV-RC2

pPLUS® AAV-RC2, which is a RepCap plasmid intended for use in the manufacture of adeno-associated virus vector 2 (AAV2), is the first in the company’s planned series of launches this year of RepCap plasmids for AAV serotypes. “In the AAV space we will be one of very few suppliers able to provide a complete pDNA portfolio, making us a logical first call for AAV manufacturers in their projects from research to commercial use,” Roel Gordjin, the chief commercial officer of Polyplus, said in a statement.

7. UK's NICE Recommends Against Use of Exa-Cel in Draft Guidance Document

Despite the Medicines and Healthcare products Regulatory Agency's approval of sickle cell disease gene therapy exagamglogene autotemcel (exa-cel) in the United Kingdom, the UK's National Institute for Health and Care Excellence (NICE) has published a draft guidance document recommending against the use of exa-cel, stating concerns related to long-term efficacy and cost-effectiveness of the treatment.

8. Capstan Therapeutics Garners $175 Million in Series B Financing

The funds from the oversubscribed round of financing, which was led by RA Capital Management, will be used with the intent of bringing Capstan's investigational approach to in vivo engineering of CAR T-cells, CPTX2309, to a proof-of-concept clinical study in autoimmune diseases. Capstan noted some of the funds will also be used to advance other items in its targeted lipid nanoparticle pipeline.