Neurology

New data shows the ASO therapy’s benefit in older populations, following beneficial findings in patients previously treated with gene therapy.

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

The data come from 30 patients treated across 2 clinical trials and 9 patients treated in expanded access frameworks who had follow-up times ranging from 0.64 years to 12.19 years.

Review top news and interview highlights from the week ending September 1, 2023.

Data from a multiple-ascending dose study suggest the investigational agent developed by Design Therapeutics was generally well tolerated with dose-related increases in frataxin levels.

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

TSHA-102 has previously received orphan drug and rare pediatric disease designations from the agency and is being investigated in the REVEAL phase 1/2 trial (NCT05606614).

In light of the promising results, BlueRock is preparing for a phase 2 trial.

Pending approval, ARO-DUX4 will be evaluated in patients with facioscapulohumeral muscular dystrophy and ARO-SOD1 will be evaluated in patients with amyotrophic lateral sclerosis.

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

Earlier this year Bloomsbury Genetic Therapies met with the UK’s MHRA for a scientific advice meeting to discuss BGT-NPC.

The codirector of the MDA Clinic and professor of neurology at Johns Hopkins discussed the importance of connecting infants with spinal muscular atrophy to a therapy as soon as possible.

BGT-OTCD is being developed in a collaboration between Bloomsbury Genetic Therapies and University College London.

The professor of neurosurgery at Rush University Medical School discussed follow-up in the phase 1/2 trial of LCTOPC1 and further research to be done.

NGN-101 will be delivered to each participant via both intracerebroventricular and intravitreal routes on the same day in the phase 1/2 trial, with the therapy assessed for safety and efficacy.

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

The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed challenges with clinical trials in neurocognitive diseases including MPS2.

In terms of safety, Taysha stated that TSHA-102 was well-tolerated and that at 6 weeks after dosing no treatment-emergent serious adverse events had occurred.

Aspen Neuroscience noted that the study will be the US’s first multicenter phase 1/2a clinical trial for an autologous iPSC-derived therapy.

Review top news and interview highlights from the week ending August 11, 2023.

LION-CS101, the study evaluating gene therapy AB-1003, is currently recruiting patients aged 18 years through 65 years with LGMD2I/R9 who have a confirmed mutation in FKRP.

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

The initiation of dosing the second cohort in Ultragenyx’s study was recommended by the Data Safety Monitoring Board after a review of safety results from the trial’s first cohort.

Taysha Gene Therapies previously reported the dosing of the first patient with TSHA-102 in June 2023.

Nicole Paulk, PhD, the CEO, founder, and president of Siren Biotechnology, discussed research she presented at ASGCT’s 2023 conference.