News

Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.

The clinical professor of neurology and pediatrics at Keck School of Medicine of USC also discussed current strategies with gene therapy administration.

The phase 1/2 GALILEO-1 trial has completed enrollment and a phase 3 trial is planned for 2025.

The Prescription Drug User Fee Act (PDUFA) target action date is August 4, 2024.

Deborah Phippard, PhD, the chief scientific officer of Precision for Medicine, discussed unique difficulties of designing clinical trials for PD gene therapy candidates.

The chairman and chief executive officer of NKGen discussed the potential of SNK01, the company’s autologous natural killer cell therapy, in treating PD.

Faraz Ali, MBA, the chief executive officer of Tenaya Therapeutics, discussed trends in precision medicine for cardiovascular indications.

Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.

The trial's primary endpoint is improvement on Luminance Dependent Navigation Assessment (LDNA).

First-in-human data with low dose VRON-0200 were presented at the EASL 2024 congress.

The therapy, rAAV-Olig001-ASPA, has shown positive clinical benefits in several interim updates from an ongoing, first-in-human phase 1/2 clinical trial (NCT04833907).

The associate professor of medicine at University of Colorado discussed a post hoc analysis study on subgroup analyses of liso-cel response.

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

The company has reported data in NMOSD, myasthenia gravis, and necrotizing myopathy over the last several months.

The associate professor of neurology at the Cleveland Clinic Lerner Institute discussed the challenges that will need to be overcome to apply gene therapy to a complex neurological disorder like PD.

The senior researcher at Seattle Children’s discussed advantages of engineered Tregs compared with natural Tregs.

Cartesian also announced that the first patient has been dosed in a separate clinical trial evaluating the CAR-T therapy in systematic lupus erythematosus.

Patients in FUMANBA-2 received eque-cel after being ineligible for ASCT after 4 cycles of induction therapy.

The associate professor of neurology at the Cleveland Clinic Lerner Institute pointed out that genetic forms of PD may be the best place to start for new therapeutic research.

CGTLive takes a look at the path gene therapies have blazed in leukodystrophies, the challenges they’ve faced, and the road they’ve paved.

The placebo-controlled multidose portion of the SELECT-HD trial included 23 patients with Huntington Disease in total.

The professor in residence for pediatrics at the Sickle Cell Center of Excellence at the University of California discussed gene therapy’s ongoing transformation of the treatment landscape.

Eight patients receiving either TSC-100 or TSC-101 have been disease-free for a median of 10 months of follow-up.

Various improvements were seen in 2 adult patients and 2 pediatric patients treated in separate clinical trials.

The Don and Marilyn Anderson Professor of Oncology at University of Wisconsin – Madison emphasized similar variables in the way of therapy uptake regardless of country.

Review top news and interview highlights from the week ending June 28, 2024.

The FDA has requested limited additional CMC information.

Faraz Ali, MBA, the chief executive officer of Tenaya Therapeutics, discussed the company’s decision to supplement the gene transfer programs in its pipeline with gene editing programs.