New York has added an­oth­er biotech start­up to its grow­ing ros­ter for the Big Ap­ple hub.

Samie Jaf­frey

The com­pa­ny is Gotham Ther­a­peu­tics, which now has a $54 mil­lion launch round to back its work on a plat­form built on the in­sights gleaned by Samie Jaf­frey, an in­ves­ti­ga­tor at the Weill Med­ical Col­lege of Cor­nell Uni­ver­si­ty who has spe­cial­ized in post-tran­scrip­tion­al mR­NA mod­i­fi­ca­tions.

Lee Babiss

The goal at Gotham is to cre­ate new drugs that can drug key pro­teins which mod­u­late mes­sen­ger RNA, with po­ten­tial­ly a big im­pact on a slate of dis­eases. Can­cer is an ear­ly fo­cus at the ear­ly-stage com­pa­ny, along with au­toim­mune dis­eases as well as a run at neu­ro­sciences, but CEO Lee Babiss is re­luc­tant to go in­to much de­tail about the specifics of their work.

That’s al­so some­thing you can ex­pect to see in a start­up, where no one likes to get in­to the weeds on drug pro­grams, or de­vel­op­ment time­lines. But the sci­ence de­serves a close look.

“While aca­d­e­m­ic re­search and the phar­ma­ceu­ti­cal in­dus­try fo­cused ini­tial­ly on mod­i­fi­ca­tions of DNA, a grow­ing body of ev­i­dence in­di­cates that mR­NA mod­i­fi­ca­tions help de­ter­mine to which de­gree genes are trans­lat­ed in­to pro­teins. RNA mod­i­fi­ca­tions and their as­so­ci­at­ed pro­tein com­plex­es there­fore rep­re­sent an un­tapped fron­tier that could yield new ther­a­peu­tic ap­proach­es,” says Jaf­frey.

In Babiss the ven­ture back­ers found some­one with a deep lev­el of ex­pe­ri­ence in R&D. He is the for­mer pres­i­dent of phar­ma re­search at Roche and was al­so the CSO at PPD, a big CRO.

That will help. Gotham is keep­ing the staffing lev­els low, with 6 full timers on the pay­roll. That might ex­pand a lit­tle, but Babiss wants much of the heavy lift­ing to be done by con­tract work­ers, keep­ing the team at Gotham — which in­cludes a sub in Mu­nich — at a vir­tu­al lev­el.

Seed in­vestor Ver­sant Ven­tures led the round, along with For­bion and GSK’s SR One. The syn­di­cate al­so in­cludes Cel­gene and Alexan­dria Ven­ture In­vest­ments.

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.