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Spotlight on Gene Therapy: finding a Cure

Mueller Lab spun off Apic Bio

Posted On: Tuesday, August 22, 2017 Posted By: Florie Borel Tags: Adeno-associated virus, Alpha-one antitrypsin, Gene Therapy, In the media

Dr. Mueller and the Mueller Lab for Gene Therapy are happy to announce the creation of Apic Bio, a preclinical stage gene therapy company.

Apic Bio Mueller Lab

The newly formed company leverages its proprietary platform to advance therapies to treat rare diseases with complex mechanisms. Its lead product, APB-101, is targeted at alpha-1 antitrypsin deficiency. Apic Bio raised investments from The Alpha-1 Project (TAP), which is the venture philantropy arm of the Alpha-1 Foundation, and from a private investor with the disease.

Dr. Mueller, Co-founder and Chief Scientific Officer of Apic Bio said: “We are encouraged by the feedback that we have received during our pre-IND meeting with the FDA that there is a clear path for us to conduct a first-in-human Phase 1/2 clinical study. Furthermore, we are very much looking forward to demonstrating the benefit of APB-101 to patients that have been living with alpha-1 and have had very little hope for a cure. Our data suggests this is a ‘liver sparing’ approach for gene augmentation which may exceed the therapeutic and safety margins when compared to a strict gene augmentation without gene silencing that may exacerbate the underlying liver disease.” 

Apic Bio screenshot

See below a selection of the press coverage of the Apic Bio launch.

Boston Globe "For this investor, it’s a life-and-death venture" 08/21/2017

Alpha-1 Foundation "Alpha-1 patient and The Alpha-1 Project join forces to find a new therapy" 08/22/2017

Business Wire "Apic Bio Launches to Advance First-in-Class Gene Therapy for Treatment of Alpha-1 Antitrypsin Deficiency (Alpha 1) --The Alpha-1 Project (TAP) leads syndicate of investors for seed round financing" 08/22/2017