PR001 utilizes AAV9 to deliver a healthy copy of the GBA1 gene to the brain. The company is currently dosing patients in a phase I/II clinical trial of PR001 and expects to provide biomarker and safety analysis on a subset of patients by mid-2021. They also expect to initiate enrollment in a phase I/II trial of PR001 for type 2 Gaucher disease in the fourth quarter of 2020 and anticipate an update on the trial in 2021.

PR001 has previously been granted fast track designation for both indications and rare pediatric disease and orphan drug designation for Gaucher disease by the U.S. Food and Drug Administration.

Improving the safety of gene therapies 2 different ways
Two groups of researchers have developed unique approaches to overcome the limitations of delivery of gene editing therapeutics. In a pair of new papers,...

UCB expands gene therapy capabilities
UCB has acquired Belgium-based Handl Therapeutics and formed a new collaboration with Florida-based Lacerta Therapeutics to accelerate the company's gene...

AGTC touts positive early clinical results for gene therapy
Applied Genetic Technologies (AGTC) has reported positive data from its ongoing phase I/II clinical trial evaluating its adeno-associated virus (AAV)-based...

Sensorion, Novasep ink gene therapy agreement
Sensorion and Novasep have signed an agreement for the manufacturing of adeno-associated virus vectors covering a gene therapy program that targets OTOF,...

Bayer acquires Asklepios to advance gene therapies
Bayer has agreed to pay $2 billion to acquire Asklepios BioPharmaceutical, a U.S.-based biotechnology company specializing in gene therapies.