FDA Clears AskBio’s AAV Gene Therapy for Muscular Dystrophy to Enter Phase I/II

FDA_Jason Armond / Los Angeles Times via Getty

Jason Armond / Los Angeles Times via Getty Images

A company known as AskBio would naturally be expected to have all the answers – and this morning, the North Carolina-based biotech announced that it had been cleared by the U.S. Food and Drug Administration (FDA) to begin testing its therapeutic candidate for a rare, genetic form of muscular dystrophy (MD). 

Asklepios BioPharmaceutical, Inc., an independently operated subsidiary oBayer AG, is developing a pipeline of adeno-associated virus (AAV) gene therapy candidates to erase genetic diseases like Pompe disease, Parkinson's disease, congestive heart failure, and of course, MD.

LION-101 is a novel recombinant AAV being developed as a one-time intravenous infusion for the treatment of adult and adolescent patients with Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9).

LGMD2I is a group of disorders causing weakness and muscle wasting in the arms and legs, particularly the voluntary muscles around the hips and shoulders. Symptoms generally strike in late childhood and gradually worsen over the next 23-26 years until patients are often confined to a wheelchair for mobility. There is currently no cure, and all available treatments are aimed at managing the symptoms. 

“Clearing of the LION-101 IND to proceed underscores our commitment to address unmet needs in musculoskeletal disorders, where very few or no treatments exist today, while we continue to advance our broad therapeutic pipeline of novel AAV gene therapies," said AskBio chief executive officer and co-founder, Sheila Mikhail. 

In preclinical research that led to the approval of the investigational new drug application (IND), LION-101 demonstrated a high potential to advance to the clinic.

“In preclinical mouse models, LION-101 therapy demonstrated both dose-dependent efficacy and tolerability, providing a clear approach to study this novel AAV vector in clinical trials," said AskBio President of Therapeutics, Katherine High, MD. 

“Currently there are no approved therapies for LGMD2I/R9, and with limited treatment options that only address symptoms of the disease, the patient burden is profound. We are excited to begin clinical trials with this novel therapy, and to hopefully bring a new therapeutic option to patients and families in the LGMD2I/R9 community living with this devastating disease."

The company plans to commence dosing in the phase I/II trial during the first half of 2022.

AskBio was co-founded in 2001 by AAV and gene therapy pioneer, R. Jude Samulski, who in 1984, cloned the virus DNA into a bacterial plasmid, laying the foundation for the only two currently FDA-approved AAV gene therapies, Luxturna for a rare inherited retinal dystrophy, and Zolgensma for spinal muscular atrophy. Samulski currently serves as AskBio’s chief scientific officer. 

The FDA’s IND approval for LION-101 marks the fifth pipeline program AskBio has advanced to Phase I/II clinical development. These include AAV2/8-LSPhGAA, which is being studied for Pompe disease, an often fatal metabolic disorder, and AAV2-GDNF, which is being evaluated for Parkinson’s. 

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