Atamyo Therapeutics has dosed the first subject in a Phase I/II clinical trial of ATA-100 to treat FRKP-related limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).
The multicentre, two-stage trial underway will assess the safety, pharmacodynamic, efficacy, and immunogenicity of an intravenous dose of ATA-100 in these patients.
It will have an open-label dose escalation phase (Stage 1) and a double-blind, placebo-controlled, randomised phase (Stage 2).
Atamyo Therapeutics chief medical officer Dr Sophie Olivier said: “After the first patient dosed in Copenhagen, we are now expecting recruitments at the two other approved clinical sites [Paris, France, and Newcastle, UK] to complete enrolment of the dose escalation phase (Stage 1) of the study.
“For Stage 2, after dose selection, we plan to open additional clinical sites in Europe and in the United States.”
A one-time gene replacement therapy, ATA-100 is a single-dose, adeno-associated virus (AAV) vector with the human FKRP transgene.
The therapy has obtained Orphan Drug Designation from the European Medicines Agency and the US Food and Drug Administration.
Atamyo Therapeutics CEO and co-founder Stephane Degove said: “This is an exciting milestone for our company but most importantly, if this clinical trial is successful, it could have a life-changing impact on patients affected by LGMD-R9.”
A rare genetic ailment, LGMD2I/R9 is caused by mutations in the FKRP-producing gene.
There are no curative therapies for LGMDR9 and its symptoms usually occur in late childhood or early adulthood.
Patients experience progressive muscular weakness that could result in a loss of ambulation and are also prone to myocardial dysfunction and respiratory impairment.
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