Asklepios BioPharmaceutical, a company focused on gene therapies, has announced that the first patient has been dosed in its phase 1/2 LION-CS101 clinical trial.
The research is evaluating the company’s AB-1003, a novel investigational FKRP gene replacement therapy. It involves patients with limb-girdle muscular dystrophy (LGMD) type 2I/R9.
LGMD type 2I/R9 is a form of the condition caused by changes in the FKRP gene and is linked to the weakness and wasting of arm and leg muscles. People with LGMD type 2I/R9 typically notice symptoms in late childhood, at around 11 years of age. These can include loss of mobility and impaired heart and lung function.
Nicholas Johnson, principal investigator, explained: “While the inherited nature of limb-girdle muscular dystrophy means those with the FKRP gene mutation can’t produce a normal FKRP protein for physiological muscle function, AB-1003 is designed to introduce the normal FKRP gene into the muscle and express a normal protein, and it has shown promise in restoring normal FKRP protein function in muscle in preclinical studies performed in mouse models of LGMD.
“This trial is the first step toward evaluating the safety of AB-1003 and assessing the potential that AB-1003 has to improve the lives of patients with this serious, inherited ultra-rare condition.”
Jude Samulski, co-founder and chief scientific officer of AskBio, reflected: “With our knowledge and expertise, we are working to harness the power of gene therapies as potential treatments for many of the world’s most devastating diseases.”
He concluded: “Our investigational therapy for limb-girdle muscular dystrophy is one of these and an important part of our portfolio, which also includes clinical stage investigational therapies for congestive heart failure, Huntington’s disease, multiple system atrophy, Parkinson’s disease, and Pompe disease.”
AB-1003 is manufactured by AskBio’s independently operated subsidiary, Viralgen, and has been granted fast track designation by the FDA and orphan drug designation by the European Commission. Recruitment for the LION-CS101 study continues.
LGMD type 2I/R9 remains a rare disease and is estimated to affect more than 5,000 people in the US and EU
by John Pinching
Source: pharmatimes.com
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