Neurology

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

Neurogene pointed out that the SAE is consistent with the known risks of AAV vector-based gene therapy.

Chris Wright, MD, PhD, the chief medical officer and head of translational research at Ring Therapeutics, discussed research presented at ASGCT 2024.

Review top news and interview highlights from the week ending November 8, 2024.

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

R. Nolan Townsend, Sandi See Tai, MD, and Kim G. Johnson, MD, discussed Lexeo Therapeutics’ LX1001 gene therapy trial that demonstrated promising safety and biomarker effects in patients with early-stage Alzheimer disease.

The noninvasive gene therapy exceeded the 30% efficacy threshold for normalizing GCase activity across all doses.

LX1001's safety profile was characterized as well-tolerated with no amyloid-related imaging abnormalities reported.

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

Review top news and interview highlights from the week ending November 1, 2024.

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

William Chou, MD, president and chief executive officer of Passage Bio, discussed feedback from a recent Type C meeting with the FDA.

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

Furthermore, the majority of patients treated with the gene therapy showed improved myelination as measured by T-2-weighted MRI.

Melissa Spencer, PhD, spoke about the emerging role of adeno-associated virus as a delivery system for gene therapies targeting muscular dystrophies, and the challenbges associated with them.

Sharon Hesterlee, PhD, the chief research officer of the Muscular Dystrophy Association discussed the upcoming MDA Clinical and Scientific Conference, which will be held March 16-19, in Dallas, Texas.

William Chou, MD, president and chief executive officer of Passage Bio, discussed feedback from a recent Type C meeting with the FDA.

Review top news and interview highlights from the week ending October 25, 2024.

Crystal Proud, MD, the director of neurology and neuromuscular medicine at the Children’s Hospital of the King’s Daughters in Norfolk, Virginia, spoke about the enhancements to newborn screening for the disease.

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

For the patients treated at the high dose, an average improvement of 18 points from baseline was observed in UPDRS Part 3 “off” medication score at 26 weeks posttreatment.

Review top news and interview highlights from the week ending October 18, 2024.

The phase 1/2 EPISOD1 study, which is taking place in the US, will provide participants with a short immunosuppression regimen before administration of the gene therapy.

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

Benitec reported that the first patient showed a 35% reduction, constituting clinically meaningful improvement, in total score on the Sydney Swallow Questionnaire.

The REVEAL adolescent and adult trial and the separate phase 1/2 REVEAL Pediatric Study are both evaluating the gene therapy TSHA-102 in Rett syndrome populations.

Review top news and interview highlights from the week ending October 11, 2024.

Paul Melmeyer, MPP, the executive vice president of public policy & advocacy at MDA, discussed the growing interest in gene therapy for the annual meeting.

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

As cell therapy investigations mature in the field of AD, some programs show promising signs of efficacy.