ASGCT: Rocket Ready to Launch, Mustang Bio’s Successful Trial and Voyager’s Early Data

Gene Therapy_Compressed

The 25th Annual Meeting of the American Society of Gene and Cell Therapy wrapped up on Thursday, bringing forth a host of new data for promising genetic therapeutics across a spectrum of diseases. Read on for highlights from the meeting’s final day.

Rocket Pharmaceuticals Set to Launch in Rare Immune Disorder

Rocket Pharmaceuticals presented positive top-line safety and efficacy data from its Phase II trial of RP-L201 for the treatment of Leukocyte Adhesion Deficiency-I (LAD-I). LAD-I is a rare disorder in which leukocytes or the white blood cells of the immune system are defected, causing immunodeficiency. People with the condition have an increased susceptibility to bacterial and fungal infections that can be recurrent, severe and life-threatening.

In Phase I and II trials of infusion treatment with RP-L201, the therapeutic demonstrated sustained CD18 restoration, a measure of leukocyte adhesion, on more than 10% of neutrophils in all patients, and overall survival without allogeneic hematopoietic stem cell transplantation across the cohort was estimated to be 100%.

All patients treated also demonstrated a statistically significant reduction in the rate of hospitalizations and severe infections and restoration of wound repair. The safety profile was highly favorable with no serious adverse events having been reported.

RP-L201 is a gene therapy that delivers a functional copy of the ITGB2 gene, which encodes for the beta-2 integrin component CD18, a protein that facilitates leukocyte adhesion and extravasation from blood vessels to fight infections. Rocket is initiating discussions with health authorities and anticipates completing regulatory filing in the first half of 2023.

Voyager Gets Promising Early CNS Data 

Voyager Therapeutics presented data from its family of AAV9-derived Tracer capsids which demonstrated cross-species central nervous system (CNS) transduction. It also presented preclinical data demonstrating a potential to ameliorate diseases in models of GBA1, tauopathies and SOD1 amyotrophic lateral sclerosis (ALS).

Voyager’s Tracer capsids are optimized to deliver diverse therapeutic payloads to the CNS to improve gene expression in the brain. New data from the company demonstrates that its newly identified capsid VCAP-102 performed at 50-fold better transduction in mice and 60-fold better transduction in non-human primates versus traditional AAV9 capsids, indicating the capsid’s potential ability to be used in humans.

Voyager also presented preclinical data demonstrating improvements in disease activity across several CNS targets: GBA1, a gene relevant to Parkinson’s Disease, tauopathies, which occur in Alzheimer’s Disease, and SOD1 ALS. Preclinical data in each program ameliorated disease states in animal models.

GBA1 transgenes, delivered to the CNS via a blood-brain barrier penetrant AAV capsid, were able to deliver therapeutically relevant doses of the transgenes. vTau, the company’s anti-tau antibody, demonstrated robust efficacy and significant reduction of tau pathology in mice models. In ALS mice models, a SOD1-targeting RNAi gene therapy improved motor performance and survival.

Mustang Bio Interim Data Supports Path Forward for X-Linked Immunodeficiency

Mustang Bio announced interim Phase I/II data from its trial of MB-107 for the treatment of X-linked severe combined immunodeficiency (XSCID). XSCID is an immunodeficiency disorder in which the body produces very few T cells and natural killer cells, making those diagnosed prone to severe and life-threatening bacterial and fungal infections.

Treatment with MB-107 resulted in complete hematopoietic recovery for all infants treated. Additionally, 15 of the 23 patients treated were able to discontinue intravenous immunoglobulin treatment and 14 patients have been successfully immunized.

The treatment was also well-tolerated with severe adverse events occurring in three patients which were all able to be resolved. Researchers noted that the therapy has allowed patients to develop a functional immune system. MB-107 is a lentiviral vector that targets IL2RG, a gene that is implicated in immune system functioning.

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