CHDI, a nonprofit foundation pursuing treatments for Huntington’s disease (HD), awarded Isis Pharmaceuticals up to $9.9 million for the discovery and development of an antisense drug for the treatment of HD. The funding builds on an earlier successful collaboration.
The backing will be provided over three years to identify and conduct IND-enabling studies on a candidate targeting the huntington gene. Mutations in the huntington gene are responsible for the production of the abnormal Huntington protein, which ultimately leads to onset and progression of the neurodegenerative disease.
Upon completion of IND-enabling research, Isis and CHDI will partner to ensure that any resulting drugs will be broadly available to HD patients. Isis has the right to continue clinical development of those drugs arising from the collaboration.
Under the previous research contract with CHDI, Isis says that its scientists demonstrated a reduction in Huntington protein expression in both brain and peripheral tissues of normal mice using Isis’ second-generation antisense oligonucleotides. In addition, the inhibition of normal Huntington expression was reportedly well-tolerated. These positive results led to the current expansion of the alliance to identify a drug development candidate for the treatment of HD.