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The professor at Ohio State University and University of California – San Francisco discussed investigations with different delivery methods of GDNF gene therapy.

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

Mary “Nora” Disis, MD, director of University of Washington Medicine’s Cancer Vaccine institute, discussed past and current clinical trials for investigational CAR-T therapies in ovarian cancer.

The director of therapeutic genome engineering, St. Jude Children’s Research Hospital discussed challenges with performing prime editing in cells.

The chief executive officer of Lineage Cell Therapeutics discussed findings related to its cell therapy OPC1 that the company presented at the first Annual SCI Investor Symposium.

The cofounder and chief executive officer of NKILT Therapeutics discussed unique advantages of the CIR platform.

Mary “Nora” Disis, MD, director of University of Washington Medicine’s Cancer Vaccine institute, discussed research she coauthored that was presented at ASCO’s 2023 conference.

The 2 patients were among 6 who have received treatment in Vertex’s trial so far and were the only 2 evaluable for the primary efficacy end point.

The chief medical officer of Trisalus Life Sciences discussed studies evaluating the company’s PEDD system.

Review top news and interview highlights from the week ending June 30, 2023.

The director of research and development at Teknova discussed the company’s recent announcement of a new line of products intended to streamline process development in AAV gene therapy manufacturing.

Five patients in the cohort achieved this milestone and Sernova noted a sixth patient in the cohort is still awaiting assessment of islet graft function.

Taysha also provided updates on its gene therapy program for Rett syndrome.

The FDA’s decision to approve this first gene therapy in hemophilia A was based on data from the phase 3 GENEr8-1 clinical trial.

Benitec Biopharma is planning to initiate a phase 1b/2a clinical trial to evaluate BB-301.

Detailed topline data will be presented at the 2023 International Congress on Parkinson’s Disease and Movement Disorders in August.

With an original PDUFA date in August 2021, the decision is a long time coming.

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

The vice president of business development and alliances at Prescient Therapeutics discussed advantages of the OmniCAR, SpyTag, and SpyCatcher technologies.

Updated data are from a total of 36 patients treated in a phase 1/2 study as of June 2023.

The director of research and development at Teknova discussed the company’s new screening kit and its potential to speed up AAV gene therapy development.

In addition to the BLA acceptance, Pfizer noted that a European MAA for the gene therapy has been accepted by the EMA.

Susan Bal, MD, assistant professor of medicine at University of Alabama – Birmingham, discussed new data on BMS-986393.

Among 4 patients who were treated at the highest dose level evaluated using a manufacturing process referred to as Process A, all 4 patients achieved responses.

The scientific cofounder and chief executive officer of Alloplex Biotherapeutics discussed progress of the phase 1 trial and possible future evaluations of the therapy.

Jason Westin, MD, FASCP, the director of the Lymphoma Clinical Research Program at University of Texas MD Anderson Cancer Center, discussed factors that limit patient access to treatment and a potential solution.

The director of the Medical Oncology Service at the National Cancer Institute discussed the START-001 trial.

The new analysis is from a phase 1 trial of Celularity’s legacy MLASC therapy.

The new data from the TRANSCEND FL and TRANSCEND NHL 001 studies are the latest in a series of positive updates regarding liso-cel that have been announced by Bristol Myers Squibb in recent months.

The director of the Lymphoma Clinical Research Program at University of Texas MD Anderson Cancer Center discussed axi-cel's safety profile and an important factor for improving access to CAR-T therapies.