The Cystic Fibrosis Foundation announced today that it has awarded more than $1.8 million to three companies for early-stage research into potential genetic therapies for cystic fibrosis as part of its Path to a Cure initiative, an ambitious research agenda to accelerate treatments and drug development for the underlying cause of the disease and ultimately deliver a cure.
“This funding will support critical early steps necessary for the development of genetic therapies for cystic fibrosis,” said William Skach, MD, executive vice president and chief scientific officer of the Cystic Fibrosis Foundation. “These promising programs are tackling difficult challenges such as efficient therapeutic delivery of diverse genetic cargos and evasion or modulation of the immune system’s response to gene delivery vehicles.”
The Foundation awarded more than $766,000 to Carmine Inc. to test its non-viral gene therapy program. This program plans to use extracellular vesicles – bits of cell membrane that naturally bud off from cells to form tiny particles – obtained from red blood cells to deliver healthy cystic fibrosis transmembrane conductance regulator (CFTR) genes into lung cells. The expected benefit of using this system is that it can potentially carry the full-length CFTR gene and additionally, it should not trigger an immune system response, which could limit repeat dosing of genetic therapies.
In addition to this award, the Foundation funded its first program directly aimed at reducing or eliminating the potential immune response from a viral delivery method. GenexGen Inc. was awarded nearly $595,000 to develop a drug that is designed to dampen the body’s immune system response to an adeno-associated virus (AAV). GenexGen is conducting preclinical testing on a CRISPR gene silencing approach that targets a key gene involved in the immune system with the goal of temporarily turning it off. If successful, this therapy could block the normal immune response to an AAV, creating an opportunity for repeat dosing.
Finally, the Foundation awarded Specific Biologics Inc. more than $527,000 to test a CRISPR-like gene editing approach aimed at correcting the three most common nonsense mutations in CF: G542X, R553X, and W1282X. Eventually, the company hopes to develop an approach that would work for any mutation. The funding will support preclinical testing of an inhaled drug that uses lipid nanoparticles, a chemically coated particle designed to help the gene editing molecules enter cells more easily.