as declared in Gene therapy for Duchenne muscular dystrophy just got a bit closer to being a reality today.
It is theorized that Exonics’ gene therapy could potentially treat 80% of Duchenne patients.
At WORLDSymposium in San Diego, CA, we talked with Tim Miller, PhD, CEO of Abeona Therapeutics about the gene therapy study they are conducting in patients with sanfilippo syndrome.
Latest ArticlesIn preclinical studies, Dr. Olson’s lab used adeno-associated virus (AAV) and CRISPR/Cas9 technology to identify and correct exon mutations in the dystrophin gene.
Patients with Duchenne muscular dystrophy lose the ability to walk as early as age 10 and experience life-threatening lung and heart complications in their late teens and twenties.There are an estimated 15,000 patients with Duchenne in the United States but the population has many subsets based on mutations of the dystrophin gene.
referring to CureDuchenne Ventures has been formed by CureDuchenne, which to date has funded nine research projects that have advanced into human clinical trials.
CureDuchenne says its funding has leveraged more than $1.3 billion in follow-on research investment from biopharma companies.
“We are honored to advance the groundbreaking work of Dr. Olson’s laboratory and are eager to translate this approach into an important therapy for the Duchenne community,” added Jak Knowles, M.D., managing director of CureDuchenne Ventures, and vp of medical and scientific affairs at CureDuchenne.
“This represents the next generation of potential Duchenne muscular dystrophy therapies,” Dr. Olson declared in a statement.
Dr. Knowles also serves as president and interim CEO of Exonics, which bases its corporate office in Boston and conducts research activities in Dallas.
referring to Exonics’ CRISPR/Cas9 technology is a potential one-time treatment that would make a permanent correction of the mutation that causes Duchenne.
About Exonics TherapeuticsExonics Therapeutics is advancing gene editing technologies like CRISPR/Cas9 to permanently correct the majority of Duchenne muscular dystrophy mutations.
“Exonics’ CRISPR/Cas9 technology has the potential to dramatically improve the lives of those who live with Duchenne.
About CureDuchenne VenturesCureDuchenne Ventures LLC collaborates with pharmaceutical and biotechnology companies to facilitate the development of drugs to treat Duchenne muscular dystrophy.
CureDuchenne Ventures LLC was formed by CureDuchenne, a national nonprofit that has funded nine research projects that have advanced to human clinical trials.
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