Amicus Therapeutics is launching a next-generation genetic medicine company called Caritas Therapeutics through a merger deal with ARYA Sciences Acquisition Corp IV, a special purpose acquisition company sponsored by Perceptive Advisors.

The U.S. Food and Drug Administration approved Nexviazyme (avalglucosidase alfa-ngpt) for the treatment of patients 1 year of age and older with late-onset Pompe disease, a progressive and debilitating muscle disorder that impairs a person’s ability to move and breathe.

Amicus Therapeutics announced topline results from the Phase III PROPEL pivotal trial of AT-GAA (cipaglucosidase alfa and miglustat) for late-onset Pompe disease.

Shares of Avrobio were up in trading after the company posted positive clinical data from gene therapy trials in three different rare lysosomal diseases: Fabry, Gaucher type 1 and cystinosis.

Data with Sanofi’s investigational enzyme replacement therapy avalglucosidase alfa in late-onset Pompe disease and infantile-onset Pompe disease will be featured as platform and poster presentations at the 17th annual WORLDSymposiumTM, to be held February 8-12, 2021.

The U.S. Food and Drug Administration granted various designations for Sanofi’s avalglucosidase alfa for Pompe disease, Sanofi’s rilzabrutinib for immune thrombocytopenia, and ViiV Healthcare’s cabotegravir for HIV prevention.

Bayer is making a big bet on gene therapy with the acquisition of North Carolina-based Asklepios BioPharmaceutical (AskBio).

Amicus Therapeutics announced positive interim results from the company’s CLN6 Batten disease gene therapy program.

Cranbury, N.J.-based Amicus Therapeutics and the Perelman School of Medicine at the University of Pennsylvania are expanding their gene therapy collaboration.

Asklepios BioPharmaceutical (AskBio) secured a $235 million investment from Vida Ventures and TPG Capital.