Verve pauses gene editing trial
02 Apr 2024
Gene TherapyClinical StudyIND
Verve Therapeutics has paused enrollment in its Heart-1 clinical trial for VERVE-101, a cardiovascular disease gene editing treatment, due to a safety concern after a participant experienced temporary health issues.
The Boston-based biotech revealed this week that the patient, who received a 0.45 mg/kg dose, experienced temporary elevated ALT (liver enzyme) levels and low platelet count, although they showed no symptoms.
VERVE-101 has been fraught with regulatory hurdles. In November 2022, the FDA placed a hold on its IND application for its single-course, in vivo liver gene editing treatment targeting heterozygous familial hypercholesterolemia (HeFH), a genetic disorder causing high low-density lipoprotein cholesterol levels.
A month later, Verve received a clinical hold letter from the FDA explaining the hold, in which the agency asked for more details on accidental germline editing risks, potency differences between human and non-human cells, and off-target effects in non-hepatocyte cell types. To address the FDA's concerns, Verve submitted interim clinical data from its ongoing Heart-1 trial and addressed preclinical questions. By October 2023, the FDA had lifted the clinical hold, allowing Verve to proceed with U.S. trials for VERVE-101.
Now, while Verve investigates the recent lab abnormalities in VERVE-101 and works with regulatory authorities to define a path forward, the company is turning its attention to VERVE-102, a different PCSK9 gene editing treatment, with regulatory approval for trials in the U.K. and Canada expected to come in the second quarter of 2024.
For more details,please visit the original website
The content of the article does not represent any opinions of Synapse and its affiliated companies. If there is any copyright infringement or error, please contact us, and we will deal with it within 24 hours.
Indications
Targets
Hot reports
Get started for free today!
Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.
Start your data trial now!
Synapse data is also accessible to external entities via APIs or data packages. Leverages most recent intelligence information, enabling fullest potential.