Matt Hoffman

M. Peter Marinkovich, MD, and Alfonso L. Sabater, MD, PhD, offered their respective perspectives on the recent FDA approval of the topical and redosable gene therapy for DEB.

The Krystal Biotech treatment, marketed as Vyjuveck, is now the first topical gene therapy for patients with DEB—marking a milestone in the paradigm of care.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The single-administration allogeneic cell therapy has shown positive safety—and signs of efficacy—in preliminary data from the first 2 patients dosed with the Neurona Therapeutics’ product.

The CAR-T therapy reduced disease progression risk by 74%, according to a data leak reported by STAT News. The data are expected to be presented on May 11 at the 2023 European Hematology Association Congress.

Atsena Therapeutics’ AAV gene therapy is being evaluated in 15 patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D across 5 cohorts of various doses. Additional data are expected to be presented later this year.

The rolling BLAs also included requests for priority review of the CRISPR/Cas9 gene-edited therapy, and is supported by data from the phase 3 CLIMB-111 and CLIMB-121 studies, as well as the long-term follow-up study CLIMB-131.

Precigen’s investigational UltraCAR-T therapy is being assessed in a dose-escalation and -expansion study that will include patients with solid tumors, chronic lymphocytic leukemia, mantle cell lymphoma, acute lymphoblastic leukemia, and diffuse large B-cell lymphoma.

The Delphi panel considerations were informed by safety data from 3 clinical trials of the therapy, and included vomiting, myocarditis, acute liver injury, and immune-mediated myositis.

The Amplo Biotechnology gene therapy showed positive findings in mouse models, suggestive of the therapy’s development for use in a clinical setting. The AAV therapy delivers DOK7 gene, which has shown benefit in neuromuscular disorders.

Presented at MDA 2023, the gene therapy (also known as delandistrogene moxeparvovec) showed significant improvements on NSAA total score, 10-meter walk/run scores, and time to rise scores relative to an external control cohort.

Ahead of the May 29, 2023, action date, the FDA is now seeking to hold an advisory committee meeting on Sarepta’s investigational agent SRP-9001 (also known as delandistrogene moxeparvovec) for the treatment of Duchenne muscular dystrophy.

The investigator-initiated, single-arm, open-label trial will follow 20 patients with high-risk LBCL for 2 years, with a primary outcome measure of complete response rate per Lugano classification.

The full dataset, published in the New England Journal of Medicine, reveals the newly approved hemophilia B gene therapy reduced annualized bleeding rates and improved the need for factor IX infusions.

The study of VX-264, a stem cell-derived, fully differentiated pancreatic islet cell therapy, is already ongoing in Canada. It is expected to kick off in the United States in the first half of 2023.

Mesoblast’s allogeneic off-the-shelf therapy showed long-term benefit in supporting data among children with steroid-refractory acute graft versus host disease. The FDA has set a PDUFA action date of August 2, 2023, for its review.

Ultragenyx’s self-complementary AAV9-based gene therapy reduced levels of relevant CSF biomarkers, as well as improvements in neurocognitive assessments and behavioral domains, among patients without neurodegeneration.

Rajesh Pahwa, MD, FANA, FAAN, discussed balancing hope with current challenges with stem cell approaches in PD.

Shephard Mpofu, MD, part of the Novartis Gene Therapies leadership, shared his thoughts on the latest phase 3 data on onasemnogene abeparvovec (Zolgensma) from the SPR1NT trial.

Data from a phase 1/2 trial of AMT-130 supports continued enrollment for a higher-dose cohort of 6 x 1013 vg.

In an AAN plenary talk, Mark H. Tuszynski, MD, PhD, detailed the work he and colleagues have done to push stem cell therapy from the lab to the clinic to improve care for spinal cord injury.

The 2-stage study is an open-label, 2-stage clinical trial designed to evaluate safety and dose-escalation (stage 1) and safety and efficacy (stage 2) of surgically delivered AXO-AAV-GM2.

Neurocrine Biosciences and Voyager Therapeutics have terminated the Parkinson disease portion of their partnership, ending development for VY-AADC, also known as NBIb-1817.

Ronald Crystal, MD, chairman, Department of Genetic Medicine, Weill Cornell Medicine, discussed the promise of gene therapy for patients with Alzheimer disease and related disorders.

Patients treated with Axovant’s gene therapy AXO-Lenti-PD reported improvements of 40% in UPDRS-III scores, and 71% improvement in activities of daily living.

Solid Biosciences noted that its response to the agency requests for further information about manufacturing as well as updated safety and efficacy data on all dosed patients led to the lift.

Atara Biotherapeutics’ allogeneic EBV-targeted T-cell therapy, ATA188, is well-tolerated in phase 1, with a dose identified for the randomized placebo-controlled portion of the study.

Three-year data from a phase 1 study of Voyager Therapeutics’ VY-AADC01 suggest it is safe and offers potential benefits for patients with Parkinson disease.

Data show the therapy’s positive safety and expression results from a small number of clinical trial participants with limb-girdle muscular dystrophy type 2E out to 1 year.

Preliminary data from the ASPIRO study of Audentes Therapeutics resamirigene bilparvovec gene therapy suggests it is safe with improvements in neuromuscular and respiratory function.