MM-A01-01

While gene therapies were traditionally thought of as one-and-done treatments, scientists now see a benefit in redosing patients down the road. The only problem? Some patients mount an immune response to the adeno-associated viruses (AAV) used to deliver the therapies. It’s one of gene therapy’s most pressing issues, and a raft of companies are working on non-viral delivery methods to get around it. But Selecta Biosciences thinks it’s found another way to tamp down the body’s immune response — and Takeda is ready to dole out more than $1 billion to get in on the action. Takeda is putting down an undisclosed amount upfront and more than $1.1 billion in biobucks to license Selecta’s ImmTOR platform for its two undisclosed gene therapies for lysosomal storage disorders, the companies said on Monday. Selecta’s platform makes use of a biodegradable nanoparticle that encapsulates the immunosuppressive drug rapamycin, and is designed to be given in conjunction with gene therapies to induce antigen-specific immune tolerance. “Most of the safety events [in the field] have been seen at the higher doses,” CEO Carsten Brunn told Endpoints News. “So if you can give a lower dose or multiple lower doses, that could really transform the treatment regimen.” Brunn declined to comment on Takeda’s specific targets or their stage of development. But he did say that Selecta filed an IND last week for its lead in-house gene therapy program, a treatment for methylmalonic acidemia (MMA) called MMA-101. Right behind that program is a treatment for ornithine transcarbamylase deficiency (OTCD), which Brunn said should be ready for an IND by the end of next year. Back in April, AskBio hit the exit on a licensing deal for MMA-101 after finding fault with a contract manufacturer’s work. Brunn said the problem was a fill-finish issue, which has since been resolved. Selecta is far from alone in the hunt for gene therapy 2.0. Anjarium Biosciences and GenEdit both unveiled hefty Series A rounds last month to support their non-viral approaches. GlaxoSmithKline vet Brian McVeigh launched Code Biotherapeutics just a few months ago to find a non-viral delivery method. And Generation Bio closed a $230 million IPO last June to fund its preclinical push to treat diseases of the liver and retina. “Selecta’s ImmTOR platform is designed to mitigate unwanted immune responses allowing for redosing, which could have broad applicability across our gene therapy programs for a range of diseases,” Madhu Natarajan, head of Takeda’s rare diseases drug discovery unit, said in a statement.