Gene Therapy Trial for Rare Form of MD Planned for Next Year

Marisa Wexler MS avatar

by Marisa Wexler MS |

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gene therapy LION-101

Asklepios BioPharmaceutical (AskBio) is planning to launch a Phase 1/2 clinical study to evaluate LION-101, its investigational gene therapy for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).

Dosing in the trial is expected to start in the first half of next year.

“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,” Katherine High, MD, president of therapeutics at AskBio, said in a press release.

AskBio, which is a wholly owned and independently operated subsidiary of Bayer AG, had submitted an investigational new drug application, or IND, to the U.S. Food and Drug Administration (FDA) requesting permission to begin clinical testing of LION-101. The FDA has now approved the IND.

“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,” said Sheila Mikhail, CEO and co-founder of AskBio.

LGMD2I/R9 is a type of limb-girdle muscular dystrophy caused by mutations in the FKRP gene. Symptoms of the disease, such as difficulty walking, typically appear first in late childhood, and patients are usually dependent on a wheelchair to get around within 23–26 years of symptoms first appearing, according to AskBio.

“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,” High said.

As a gene therapy, the aim of LION-101 is to deliver a non-mutated version of the FKRP gene to patient’s cells. It’s designed to do this using a specifically engineered viral vector, a recombinant adeno-associated virus (AAV) — this type of virus is commonly used in gene therapies because it is relatively easy to manipulate in the lab, and it generally does not cause serious illness in people.

The therapy is designed to be administered via a one-time infusion directly into the bloodstream.

“In preclinical mouse models, LION-101 therapy demonstrated both dose-dependent efficacy and tolerability,” High said, adding that these data have provided “a clear approach to study this novel AAV vector in clinical trials.”

No details on the design of the Phase 1/2 clinical trial are available yet.