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Competitor development AAT:Apic Bio announces $40M financing to advance novel gene therapies for rare genetic disordersApic Bio, Inc., an innovative gene therapy company developing novel treatment options for patients with rare genetic diseases, announced the completion of a $40 million Series A financing led by Morningside Venture Investments, Ltd. Existing investors, The Alpha-1 Project (TAP) and A1ATD Investors, LLC also participated along with new investor, the ALS Investment Fund. The new capital will advance Apic’s APB-101, APB-102 as well as preclinical Alpha-1 Antitrypsin Deficiency (Alpha-1) and amyotrophic lateral sclerosis (ALS) programs including gene insertion of alpha-1 antitrypsin (AAT) and targeting of the C9orf72 mutation. Additionally, the new funding will support further discovery efforts leveraging the company’s proprietary silence and replace THRIVE™ platform. “We are excited about next-generation treatments for individuals with Alpha-1 and we are hopeful that this therapy will be useful in the treatment of newly diagnosed patients,” said Miriam O’Day, interim CEO of the Alpha-1 Foundation. APB-101 is a “liver-sparing” gene therapy designed as a one-time treatment for Alpha-1 patients. In pre-clinical studies, it has demonstrated the ability to reduce levels of the mutant AAT protein (Z) and at the same time program liver cells to produce the correct AAT protein (M). This announcement comes right after an active year in the gene-silencing therapy field, with the announcements by Alnylam Pharmaceuticals and by Arrowhead Pharmaceuticals, on submission for Clinical Trial Authorization (CTA) to initiate a Phase 1/2 study of ALN-AAT02 in the United Kingdom (by Alnylam), and on the completion of enrollment for Phase 1 clinical trial of ARO-AAT (by Arrowhead). Also, the U.S. Food and Drug Administration (FDA) approved the first small interfering ribonucleic acid (siRNA) treatment, in 2018. source: www.businesswire.com/news/home/20190107005364/en/Apic-Bio-Announces-40M-Series-Financing-Advance |
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