Coupling mRNA with tRNA and new delivery tech, MPM and OrbiMed help birth new $80M biotech into a slowed down world
Partners at MPM and OrbiMed were hoping David Lockhart could help them with due diligence when they brought him and his team at TranscripTx in to look at a tiny upstart spun out of the University of Texas Southwestern Medical Center in Dallas. Dubbed ReCode Therapeutics, it was developing a lipid nanoparticle (LNP) delivery platform for various genetic medicines, with a lead program in cystic fibrosis backed by the CF Foundation.
But Lockhart, the former CSO of Amicus Therapeutics and a one-time biotech entrepreneur, saw an immediate match with the mRNA and respiratory disease work that TranscripTx was doing. Since his company was also looking to raise money, wouldn’t it make sense to merge the two?
So instead of two Series B rounds, MPM and OrbiMed have now put their money behind a brand new biotech that’s taking the ReCode name and $80 million in Series A cash to hustle toward the clinic and get serious about BD — albeit at a slightly slower pace amid a pandemic.
“We closed the merger and the financing on March 6,” Lockhart told Endpoints News. “Even though in some ways that wasn’t very long ago, the world was kind of in a different place even three weeks ago.”
Colt Ventures co-led the round with OrbiMed, with participation from Vida Ventures, Hunt Technology Ventures and Osage University Partners.
The academic labs at UT Southwestern, where founders Dan Siegwart and Philip Thomas are based, have been shut down. With most of the staff working from home, the CEO often finds himself working in an empty office in Menlo Park by himself. ReCode doesn’t plan to pull the trigger on any new hires in the next few weeks.
What they are doing, though, is laying the groundwork so that they have everything lined up to go at full speed once they can, from preclinical experiments to job candidates. The scientists who are tending to crucial cell lines are also still at work while keeping their distance, which is relatively easy with a team of 12 in California and just 3 in Texas.
The original lead program at TranscripTx was designed to treat the rare genetic disorder primary ciliary dyskinesia (PCD) using an mRNA drug delivered by an LNP licensed earlier. While ReCode is moving forward with it, the team will also test other candidates using the proprietary platform in parallel. If the results pan out, ReCode will likely swap it out, Lockhart said.
Not only is the class of LNPs invented by Siegwart — who studied under MIT whiz Robert Langer — easier to make and versatile in the payload they can carry, they can also be delivered systematically then distributed to different tissue types.
There are no approved treatments for PCD, and as with CF before Vertex’s therapies came along, patients can die of respiratory failure in their 30s or 40s even on the best supportive care. And several genetic mutations are known to cause the disease.
“PCD alone is a pipeline that will be bigger than the Genzyme pipeline that made Genzyme what it was,” Lockhart said, citing the rare disease giant that Amicus was often compared to.
In CF, Vertex has made dramatic breakthroughs with its arsenal of drugs targeting mutant CFTR proteins, covering 90% of patients. ReCode’s goal is to catch the other 10%: those with a nonsense mutation that prevents a full length protein from being produced in the first place. With the LNP tech, its therapy ushers in a piece of transfer RNA — a bridge between messenger RNA and protein synthesis — to the premature stop codon and alters the cell machinery, coaxing it to make a functional version of CFTR.
PTC Therapeutics has taken a similar read-through approach with its small molecule drug, but while ataluren has demonstrated (controversial) effect in Duchenne muscular dystrophy, it failed in CF.
“They’re actually sort of sister diseases in some ways,” he added. “The same doctors that treat PCD treat people with CF.”
Beyond these initial programs, for which clinical trials are still expected in 2021, there’s plenty of room for licensing and collaborations. It was a key topic for the new board of directors, now comprising OrbiMed’s Peter Thompson, MPM’s Ed Hurwitz, Vida Ventures’ Helen Kim, R.A. Session from BridgeBio and Michael Torres, ReCode founder and new VP of R&D.
“We had our first board meeting with the new board just on Friday,” Lockhart said. “Everyone was in a room by themselves talking into a phone.”