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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.

The cofounder and chief executive officer of Kate Therapeutics discussed research that awarded him an Outstanding New Investigator at the ASGCT 2024 meeting.

The company has begun enrolling participants in a new cohort for younger patients in the phase 1/2 AFFINITY DUCHENNE clinical trial.

Three participants with ATTR amyloidosis have received follow-on doses of NTLA-2001 with no serious complications for up to 3 years of follow-up.

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

The chief scientific officer of Precision for Medicine discussed unique difficulties of designing clinical trials for PD gene therapy candidates.

All 3 participants who have follow-up of at least 1 year have met the endpoint so far.

The company has dosed the sixth patient in the study, and expects to move onto the third cohort pending continued safety at 1 month posttreatment.

One patient is considered disease-free at 1 year posttreatment, but another patient’s disease relapsed at 5 months.

The professor from Cedars Sinai discussed further research being conducted in the field after lifileucel’s approval.

The CLARA trial has also completed enrollment of participants with corneal edema secondary to corneal endothelial disease.

The head of research at Mass General Brigham’s Cell and Gene Therapy Institute discussed work the center is engaged in with cell and gene therapy.

With the company having recently announced that it intends to expand the clinical trial with a new cohort, CGTLive® has decided to take a closer look at this ongoing study.

AMT-130 has shown some evidence of dose-dependent clinical benefits in treated study participants.

Fractyl Health is conducting IND-enabling studies for Rejuva.

Faraz Ali, MBA, the chief executive officer of Tenaya Therapeutics, discussed the company’s research on capsids, promoters, and manufacturing improvements.

For the 3 patients who were treated at the higher dose, immunohistochemistry showed that a mean of 54% of muscle fibers were expressing microdystrophin.

The chief medical officer of Encoded Therapeutics overviews the company’s timeline and strategy for its gene regulation program for Dravet syndrome.

NRTX-1001 consists of human interneurons that provide long-term secretion of gamma-aminobutyric acid, an inhibitory neurotransmitter, which is expected to repair neural networks.

Review top news and interview highlights from the week ending June 21, 2024.