The randomized, controlled clinical trial treated patients with either a high dose of MCO-010, a low dose of MCO-010, or a sham–control.

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.

RiboX stated that this is the first IND clearance the FDA has granted for any circular RNA therapy.

The attending physician at the Cancer Center at Children's Hospital of Philadelphia discussed a study she coauthored that was recently published in Blood.

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.

The company made the resubmission after reaching an accord with the FDA in a Type A meeting with the agency that took place in August 2024.

The 3 experts discussed Lexeo Therapeutics’ LX1001 gene therapy trial that demonstrated promising safety and biomarker effects in patients with early-stage Alzheimer disease.

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.

Patients with melanoma treated with ACTengine IMA203 achieved high response rates, with progression-free survival of 6 months and duration of response beyond 1 year.

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.

Participants in the trial received either a single dose of 25 mg of NTLA-2002, a single dose of 50 mg of NTLA-2002, or a placebo.

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.

The DSMB’s decision was made with reference to safety data from the GARDian trial’s phase 1 portion.

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.

The company noted that it expects to submit a BLA in the second half of next year.

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

Autologous cell and gene therapies show promise but face barriers such as high costs, limited access, and payer concerns over long-term effectiveness.

Joshua M. Hare, MD, the cofounder and chief science officer of Longeveron, discussed working to show potential benefits in a follow-up phase 3 trial.