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news.Isis Pharmaceuticals is to receive up to $9.9 million in funding from CHDI, a non-profit foundation, to aid its discovery and development of an antisense drug for the treatment of Huntington's disease, a fatal neurodegenerative disease.
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CHDI’s funding builds upon an earlier successful collaboration between Isis and CHDI, in which CHDI provided Isis with funding for studies that demonstrated the feasibility of using antisense drugs to treat Huntington’s disease (HD).
CHDI will provide funding over three years to identify and conduct IND-enabling studies on an antisense drug targeting the huntingtin gene. Upon completion of IND-enabling studies, Isis and CHDI will continue to collaborate to ensure that any resulting drugs will be broadly available to HD patients and Isis has the right to continue clinical development of those drugs arising from the collaboration.
Frank Bennett, senior vice president of research at Isis, said: “We are pleased to expand our relationship with CHDI to identify a potential new therapy for Huntington’s disease. Through several collaborative efforts, we have been focusing on the use of antisense drugs to inhibit production of central nervous system targets including Huntingtin. With ISIS 333611, our drug targeting SOD1 for a hereditary form of ALS, we have demonstrated in rodents that direct delivery of an antisense drug to the spinal fluid via an implantable pump effectively reduces SOD1 RNA and protein.”