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news.Dimension Therapeutics has secured approval from the National Institutes of Health’s Recombinant DNA Advisory Committee (RAC) for a phase 1/2 study protocol for DTX301 to treat Ornithine Transcarbamylase (OTC) deficiency.
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The RAC also issued recommendations on several design elements of the research and clinical development program.
DTX301 is an adeno-associated virus (AAV) gene therapy product being developed to offer OTC gene expression in a durable fashion, avoiding the complications related with OTC deficiency.
Preclinical trials completed till now suggest that DTX301 has the potential to be a well-tolerated, effective therapy for OTC deficiency, the most common urea cycle disorder.
OTC deficiency occurs due to a genetic defect in a liver enzyme responsible for detoxification of ammonia.
It is estimated that over 10,000 patients are affected by OTC deficiency globally, of which about 80% are classified as late-onset.
Based upon the RAC vote, the company aims to continue to go ahead in completing its IND-enabling studies to support regulatory submissions globally in the second half of this year.
Dimension Therapeutics CEO Annalisa Jenkins said: "The RAC’s unanimous approval is an important milestone for Dimension and the patient community, and represents a key step in advancing our lead IMD candidate towards the clinic.
"We are encouraged by the evolving preclinical and clinical database supporting the safety profile of the AAV vector class that has taken the field well beyond the unfortunate events nearly 20 years ago when it was confirmed that an adenovirus vector was not a viable approach that could be safely administered to humans."