Rectify, Incubated by Atlas Venture, Raises $ 100 Million to Take Drug Hunting to New Territories

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One of the quirks of the drug hunt is that finding new drugs doesn’t always mean discovering something completely new. Sometimes it’s about revisiting previously hunted lands and spotting something that was missed. Rectify Pharmaceuticals is developing the science that led to a successful lung drug franchise and taking this approach for more organs, with the liver as the first target. The young biotech company doesn’t even have its own offices yet, but it has $ 100 million to speed up its research.

The Series A funding round announced Thursday was co-led by Atlas Venture, Omega Funds, Forbion and Longwood Fund.

Rectify, which currently operates from Atlas’s incubator, based in Cambridge, Mass., Aims to treat disease by targeting transporter proteins, proteins responsible for moving substances within and within exterior of cells. The company focuses specifically on ABC transporters, a family of 48 membrane-bound proteins. Rajesh Devraj, President and CEO of Rectify and Atlas venture capital partner, said mutations in nearly half of the proteins in this class lead to diseases of the liver, lungs, gastrointestinal tract , eyes and central nervous system.

“We want to be the first company to be an expert and have in-depth knowledge of the drug from the entire class of ABC carriers,” said Devraj.

Diseases caused by mutated transporter proteins can manifest as a defect in trafficking, Devraj explained. If the protein is mutated or slightly shifted so that it has a different shape, the cellular machinery that removes damaged proteins gets rid of these mutated or distorted proteins. In other cases, when the protein is transported to the cell membrane, it does not work. In either case, a Rectify drug would correct the defect, restoring the function of the ABC protein. The company’s research identifies small molecules that can target these proteins. He calls these molecules “positive functional modulators”, or PFMs.

We already have drugs that process mutated ABC transport proteins, and successful drugs. Cystic fibrosis (CF) is caused by a genetic mutation that leads to a defective version of a CFTR, a protein important for transporting ions inside and outside cells. Defective CFTR causes mucus to build up in the lungs, hallmark of the disease. Vertex Pharmaceuticals has developed drugs that improve the function of this key protein. Kalydeco was the first of Vertex’s cystic fibrosis drugs developed to treat the mutations that cause the disease.

Rectify Founder and Scientific Director Jonathan Moore is familiar with Vertex drugs. He was a founding scientist of the Boston company, where he was heavily involved in the development of its cystic fibrosis drugs. Moore left Vertex in 2018 and then researched ABC transporters during his two years as a visiting professor at the Massachusetts Institute of Technology. Rectify was founded in Atlas about a year ago, where it is incubating. Devraj has stated that Rectify’s intellectual property is completely independent of Vertex or MIT.

While Vertex focused on CFTR, Atlas broadened the view of ABC carriers, Devraj said. There is a very large class of protein targets that can be drugged, but no one has done so yet. Reaching these targets with small molecules Rectify offers the possibility of combating unaffected diseases. The startup’s primary targets are ABC transporters which cause liver disease with little or no treatment available.

While Rectify’s research produced a main program for a liver disorder, Devraj declined to reveal it. He said the startup aims to treat rare diseases whose underlying genetic cause is understood. The list of rare liver diseases caused by transport protein problems include progressive familial intrahepatic cholestasis, a disease characterized by the inability to properly drain bile, and Wilson’s disease, which leads to the accumulation of copper in the liver and other organs.

Other biotech companies are looking for drugs that treat both PFIC and Wilson. In July, Boston-based Albireo Pharma obtained the first FDA approval for a drug that treats the pruritus, or severe itching, associated with PFIC caused by a particular genetic mutation. Mirum Pharmaceuticals, a biotechnology company based in Foster City, Calif. That received FDA approval last month for a drug that treats pruritus caused by Alagille syndrome, a rare liver disease, is also developing this drug. for PFIC, but for a different mutation than the one discussed. by Albireo. In Wilson’s disease, Alexion, AstraZeneca’s new rare disease affiliate, reported phase 3 positive data in August for its drug candidate. Alexion said he plans to submit data for regulatory review in the coming months.

Devraj said the $ 100 million funding will allow Rectify to continue to develop its technology, work on its drug candidates and advance core human proof of concept programs. He declined to offer a time frame for when Rectify expects to be ready to file for a new investigational drug. But Devraj added that the money will also allow the company to work on other programs that treat diseases beyond the liver.

“At this point, the funding of $ 100 million is aimed at obtaining the flagship product [liver disease] program in motion, and build the other pipeline programs behind it, ”Devraj said. “It’s a lot of funding that allows us to do a lot, to really focus and execute.”

Photo by Flickr user rachaelvoorhees via a Creative Commons Licence


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