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Biotech millionaire funds free drugs for ultra-rare diseases

Patients will connect to n-Lorem via academics working through the Undiagnosed Diseases Network and beyond. The foundation will identify eligible patients and Ionis will develop the ASOs, perform preclinical testing and submit Investigational New Drug (IND) applications along with investigators. To formalize the ASO development process, Ionis will need buy-in from regulators. The usual safety studies will necessarily apply, and Arthur Krieg, a cofounder of the Oligonucleotide Therapeutics Society, says the largest cost in developing therapies is toxicology studies. “This is where it will be tough for FDA and academic medical centers: how far back can we safely cut? You don’t want to endanger patients.” Given the frequently dire circumstances of ultra-rare disease patients, n-Lorem is counting on flexibility from the FDA when it comes to preclinical requirements, which would save both time and cost. But it is unclear what that flexibility would entail. Crooke’s early interactions with the FDA gave him a “general sense” that toxicology testing requirements would be manageable but expects it will be some time before he receives definitive answers from the agency.

#mediumismedicine #orphan-diseases - nature.com

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