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Apic Bio, a spin-off from the University of Massachusetts Medical School, has raised $40 million in series A financing to develop gene therapies for people with the neurodegenerative disease amyotrophic lateral sclerosis (ALS) and a rare genetic disease called α-1 antitrypsin deficiency. Apic’s approach, developed at UMass, uses adeno-associated viruses to introduce a microRNA, which silences the mutant gene, along with instructions for a new, functional gene. Apic CEO John Reilly says the funds should get the programs through Phase II clinical studies.
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