Leading-edge CRISPR biotech chooses FSHD as its first disease target
Epic Bio, based in the San Francisco Bay Area, announced that FSHD is the first disease it plans to take on with its proprietary CRISPR technology.
Founded by Stanley Qi, PhD, a co-inventor of the Nobel Prize-winning CRISPR patent held by the University of California, Epic Bio has developed a form of CRISPR that can target highly specific regions of the genome without cutting the DNA. Their technology then delivers molecules called Cas9 that modify that part of the genome in order to interfere with a disease-causing process. For example, Cas9 can be designed to activate or inactivate a gene in the target region. This makes it well suited to dial down the expression of DUX4, the gene that causes FSHD.
In laboratory experiments, the “results demonstrate the utility of our dCas to suppress DUX4 expression by up to 95%,” said Amber Salzman, PhD, chief executive officer of Epic Bio. “We view this to be a tremendous step forward to finding a one-and-done treatment for this debilitating disease.
“Following on from this, we have submitted a request to the FDA for a pre-IND meeting to align on the IND submission package and Phase 1 clinical design,” Salzman continued. IND stands for Investigational New Drug, the designation awarded by the US Food and Drug Administration when it allows a new drug to be tested in human clinical trials.
Epic Bio’s proprietary Gene Expression Modulation System (GEMS) platform “integrates its dCas proteins with customized guide RNA and modulator proteins to develop effective and safe approaches to treat diseases,” the company said. “Epic Bio’s dCas proteins are half the size of standard Cas9 proteins, thus enabling Epic Bio to deliver single AAV (adeno-associated virus) vectors to all tissues and organs.” A more efficient viral delivery system would presumably reduce the potential for toxic side effects.
In addition to its initial focus on facioscapulohumeral muscular dystrophy, Epic Bio is conducting research to address alpha-1 antitrypsin deficiency (AATD), heterozygous familial hypercholesterolemia (HeFH), as well as other indications. The company is financially backed by Horizons Ventures and other leading investors and has raised $55 million in a Series A round.
Signaling the company’s commitment to patients and families, Epic Bio served as presenting sponsor of this year’s Bay Area Walk & Roll, providing financial and logistical support as well as fielding a team of 20 employees who raised additional funds. It feels like the beginning of a beautiful partnership!
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This sounds like it has a lot of potential. The problem is that it will probably take decades to get this to those that need it the most. And those that need it the most do not have decades to wait? Also has myoaav not been vetted enough to be used to deliver this. Epic’s CEO in other press discusses derisking by using vectors already used in DMD trials but does not mention myoaav. It seems like myoaav vector to deliver this therapy would be a perfect match.
It may take decades depending on many factors but the fact that they’re in a pre IND stage of drug development tells me they’re close to doing a clinical trial which means it’ll probably be within a decade (correct me if I’m wrong) after doing quick research on drug development process. So I don’t think it’ll take “decades” but rather one decade or less.
I would not underestimate how long this will drag out. Complex mRNA Covid vaccines were developed, tested, manufactured and distributed to millions of people in months, not years or decades. Yet the trial process alone for FSHD treatments like this will almost for sure be significantly longer than a decade.
If we are lucky, Epic Bio will open this to Expanded Access before FDA approval. I am not sure how that works with gene therapies, but maybe if there is enough demand from the patients they will participate in Expanded Acess.