The biotech incubator behind Moderna will pool together its portfolio startups to work on new treatments for cystic fibrosis using up to $110 million in financing from a foundation dedicated to the genetic disease that ultimately derails one’s ability to breathe.
The Cystic Fibrosis Foundation will dole out up to that amount to Pioneering Medicines, which brings together technologies from biotechs incubated at Flagship Pioneering. One of the outfits central to the work will be Tessera Therapeutics, which snagged its own haul of $230 million back in January.
Proceeds will bankroll “multiple development candidates” through to the human proof-of-concept stage, the groups said Wednesday. The deal includes forming a separate company based on the combined work. The initial funding is $20 million, which derives from the foundation’s $500 million “Path to a Cure” program that began in October 2019.
That program has also funded the likes of Deep Science Ventures, which partnered with the foundation in June for an undisclosed amount of money to address gene therapy hurdles.
Through the Flagship pact, the new company will develop technology aimed at creating a functional CFTR protein in lung cells and a gene editing model for correcting various mutations in the CFTR gene, the foundation said. Those will be paired with a delivery method that targets the desired cells in the lung and potentially other tissues, as well.
Tessera will apply its so-called gene writing program, a form of treatment in which short or long messages are inscribed into the genome to alter the course of diseases. Tessera will use its “gene writers” to correct certain mutations in the CFTR gene and write the full length of the gene with “all-RNA therapeutic compositions,” said the biotech’s CEO, Geoffrey von Maltzahn, in a statement.
The foundation and leaders from the biotech incubator will form a joint steering committee to oversee R&D decisions.
About 10% of people with cystic fibrosis have “rare or nonsense mutations” that have no effective therapies, said Paul Biondi, president of Pioneering Medicines, in a statement.
The investigational treatment landscape for cystic fibrosis hit a snag when Arrowhead Pharmaceuticals paused a phase 1/2 trial in July due to unexpected lung inflammation in a preclinical study.