News

Review top news and interview highlights from the week ending July 7, 2023.

Among 29 patients who were treated with nusinersen in the RESPOND trial and evaluable for efficacy, most demonstrated an increase in mean total HINE-2 score from baseline.

A trial in China was initiated in December 2022 for patients with neovascular and wet age-related macular degeneration.

In terms of safety, Capricor Therapeutics’ CAP-1002 was reported to be well-tolerated.

Data from patients treated in early access programs were presented at the 9th EAN Congress.

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 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.