The FDA has re­leased a clin­i­cal hold on Pfiz­er and Sang­amo’s he­mo­phil­ia A gene ther­a­py af­ter or­der­ing the com­pa­nies to stop the tri­al last year due to con­cerns about blood clots.

Just a week af­ter Pfiz­er an­nounced the FDA had un­leashed its gene ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy from a clin­i­cal hold — which was paused as one pa­tient died and oth­ers ex­pe­ri­enced se­vere side ef­fects  — an­oth­er one of its gene ther­a­py pro­grams has been giv­en the green light to re­sume, Pfiz­er an­nounced dur­ing its Q1 earn­ings call yes­ter­day.

How­ev­er, the com­pa­ny an­tic­i­pates it won’t restart the Phase III tri­al un­til the third quar­ter of this year, Pfiz­er CSO Mikael Dol­sten not­ed dur­ing the meet­ing. In the mean­time, Pfiz­er needs to get ap­proval of their up­dat­ed study pro­to­cols from the FDA, ac­cord­ing to their Q1 earn­ings news re­lease.

Pfiz­er vol­un­tar­i­ly paused the study late last year af­ter pa­tients ex­pe­ri­enced ab­nor­mal­ly high lev­els of fac­tor VI­II, an es­sen­tial blood clot­ting fac­tor. In he­mo­phil­ia A, pa­tients lack a func­tion­al gene for mak­ing fac­tor VI­II, mean­ing that they have very low fac­tor VI­II lev­els at un­der 50%. In peo­ple with­out he­mo­phil­ia A, fac­tor VI­II lev­els usu­al­ly range be­tween 50% and 100%, but in the Pfiz­er tri­al, pa­tients had fac­tor VI­II of above 150%, putting them at risk for blood clots.

Soon af­ter Pfiz­er paused the tri­al, the FDA slapped on a clin­i­cal hold.

One pa­tient in the tri­al ex­pe­ri­enced deep vein throm­bo­sis be­low the knee, Pfiz­er not­ed in the news re­lease. That pa­tient had a his­to­ry of blood clots be­fore the tri­al be­gan, which is an ex­clu­sion cri­te­ria for par­tic­i­pat­ing in the study. How­ev­er, the pa­tient is now do­ing well, Pfiz­er said.

Now with the clin­i­cal hold gone, Pfiz­er and Sang­amo may have a chance to catch up to Bio­Marin, as the biotech plots to re­turn to the FDA with its gene ther­a­py this year. Bio­Marin’s gene ther­a­py was re­ject­ed by the FDA two years ago af­ter mul­ti­ple re­ports of fac­tor VI­II lev­els dwin­dling in pa­tients just years af­ter they re­ceived what is sup­posed to be a long-term fix.

Pfiz­er ex­pects a read­out from their he­mo­phil­ia A pro­gram some­time in the sec­ond half of 2023.

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

The European Medicines Agency said Thursday that it’s launching a new pilot program to help academic and other nonprofit researchers developing advanced therapy medicinal products, which includes cell and gene therapies.

Academics have proven to be enormously useful in feeding new products, like chimeric antigen receptor (CAR)-T cell therapies first developed by Memorial Sloan Kettering, and ushered to the market by biopharma companies. Jean Bennett, formerly with the University of Pennsylvania, also saw her research lead to the approval of gene therapy Luxturna, which Roche now owns.

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

The US Senate passed via unanimous consent on Thursday afternoon a bipartisan bill that would alter a federal mandate for animal testing on new drugs, but stops short of removing animal testing entirely.

Touted as a much-needed modernization of FDA’s rules, co-sponsor Sens. Rand Paul (R-KY) and Cory Booker (D-NJ) have said the bill will stop lots of needless suffering of animals.

Today the Senate unanimously passed my bipartisan FDA Modernization Act 2.0 to end animal testing mandates! This is an important step toward innovation and ending the needless suffering and death of animal test subjects. pic.twitter.com/vGpCCdpuOf