Sarepta Therapeutics Inc., of Cambridge, Mass., said it is offering to sell, subject to market and other conditions, $250 million in shares of its common stock in an underwritten public offering.

Orchard Therapeutics Ltd., of London, won an FDA rare pediatric disease designation to OTL-101, its lead program for the treatment of adenosine deaminase severe combined immunodeficiency, a rare inherited disorder caused by mutations in the gene encoding for the enzyme adenosine deaminase that result in a severe deficiency in white blood cells and life-threatening infections.

Janssen Sciences Ireland UC, of Cork, Ireland, a unit of Johnson & Johnson, said a regimen of two investigational long-acting, injectable formulations of HIV medicines – Janssen's rilpivirine and Viiv Healthcare's cabotegravir – given together every four or eight weeks was as effective as three-drug oral antiretroviral therapy at maintaining HIV-1 viral suppression through 96 weeks (HIV-1 RNA

PARIS – At the International AIDS Society's 2017 meeting on HIV science, data from the APPROACH vaccine trial that were presented in Monday's symposium on translational vaccinology were the most salient example of why there is what National Institute of Allergy and Infectious Diseases Director Anthony Fauci termed "a very cautious optimism" that despite decades of unsuccessful attempts, a vaccine is ultimately possible.

With Celgene Corp. gaining strategically important rights to a PD-1 inhibitor developed by Beigene Ltd. earlier this month and with activity in the PD space still revving, Cytomx Therapeutics Inc. CEO Sean McCarthy told BioWorld Insight it's important that oncology "not get too stuck [on a single drug class] and continue to ask questions" scientifically.