EPIC Bio Pte Ltd.

Decades ago, Amber Salzman remembers feeling like there was nothing she could do to help her cousin-in-law with a progressive muscle disease called facioscapulohumeral muscular dystrophy, or FSHD. She was a pharmaceutical executive who had successfully gotten a transformative gene therapy developed for her son, who was diagnosed with a deadly neurological disease, but she didn’t have a solution for FSHD because the cause of the disease was not well understood. “I’m a drug developer. I’m not a basic research scientist,” said Salzman, who is now CEO of Epicrispr Biotechnologies. “I don’t know how to fix this.” Fast forward to Wednesday. Epicrispr, which was previously more commonly known as Epic Bio, raised a $68 million Series B to study an epigenetic editing treatment in people with FSHD, a form of muscular dystrophy. The biotech received clearance from New Zealand authorities to begin a clinical trial of its experimental epigenetic editing treatment in adults with FSHD, and it plans to dose its first patient later this year. Over the last two decades, two key innovations led to this experimental therapy. First, researchers figured out the cause of FSHD — abnormal activation of a gene called DUX4 generates molecules that are toxic to the muscles. Second, as the name of the company suggests, CRISPR-based editing tools have enabled scientists to go after genetic diseases in new ways. Epicrispr was founded by Stanley Qi, an alum of Jennifer Doudna’s lab who now runs a lab at Stanford Medicine. Known as EPI-321, Epicrispr’s experimental one-time treatment is meant to silence DUX4 expression. It will be packaged and delivered via viral envelopes called AAVs. The therapy doesn’t cut DNA but rather methylates it — by adding a chemical group to silence gene expression. “What’s really exciting is we are actually going after the root cause,” Salzman said. The Series B was led by Ally Bridge Group and also included SOLVE FSHD, a venture fund started by Lululemon founder Chip Wilson, who was diagnosed with the disease. FSHD is a progressive muscle disease that primarily affects the muscles in the shoulders, arms and face. Investor interest in neuromuscular disease remains alive despite the broader downturn in the gene therapy and gene editing field. In November, Novartis bought muscular dystrophy gene therapy developer Kate Therapeutics for $1.1 billion. The same month, Sarepta made a $500 million upfront deal with Arrowhead Pharmaceuticals to work on a series of experimental rare disease treatments, including one for FSHD.

Dive Brief:Epicrispr Biotechnologies has raised $68 million in pursuit of a first-of-its-kind genetic medicine for a rare neuromuscular disorder called facioscapulohumeral muscular dystrophy.EPI-321, the startups lead program, uses CRISPR tools to stop errant expression of a gene implicated in the muscle-wasting condition. Epicrispr will start a Phase 1 trial this year in New Zealand, according to the companys Wednesday statement.The San Francisco Bay Area biotechs Series B round was led by Ally Bridge Group and involved Solve FSHD, an advocacy group formed by Lululemon Athletica founder Chip Wilson.Dive Insight:FSHD is a rare neuromuscular disorder estimated to affect about 870,000 people worldwide. The disease is characterized by progressive muscle weakness that begins in the face, back and upper arms and can leave people in wheelchairs or with debilitating pain and fatigue.Though there are no available medications for FSHD, drugmakers in recent years have zeroed in on a gene called DUX4. In FSHD, a genetic error causes DUX4 to be overexpressed, eventually resulting in muscle degeneration and atrophy. Biotech companies have been working on various ways, from small molecule drugs to gene therapies, to stop that from happening.One high-profile effort, an oral drug developed by Fulcrum Therapeutics and Sanofi, failed in Phase 3 testing last year. But other companies, including Avidity Biosciences, Novartis,Arrowhead Pharmaceuticals and Dyne Therapeutics have drugs in development as well. There are currently more than a dozen active DUX4-targeting drug programs, according to the nonprofit FSHD Society.Epicrispr says its approach is unique among that group. The company is using CRISPR tools to turn genes on or off instead of altering DNA directly. In FSHD, its harnessing CRISPR to bind a precise region of the DUX4 gene and make a chemical modification. The hope is doing so might stop expression of the encoded protein, without the health risks associated with cutting into DNA.According to Amber Salzman, the companys CEO, that strategy has shown potential in preclinical tests to impact muscle function and block the DUX4 protein from seeping out.We're going after the absolute root cause of the disease, she said. It's a really, really different approach.Epicrispr CEO Amber Salzman.Permission granted by Epicrispr BiotechnologiesSalzman has for years worked on genetic disorders as a biotech executive and patient advocate. While at GSK many years ago, her son and two nephews were diagnosed with a rare disease called adrenoleukodystrophy. She connected with prominent gene therapy researcher Jim Wilson, met several other experts in the field and started the nonprofit Stop ALD Foundation.One of Salzmans nephews died from ALD in 2004. But her son and second nephew received a treatment that was later approved as Skysona. In the meantime, Salzman worked at multiple biotech startups, including eye gene therapy developer Adverum Biotechnologies. In 2021, a recruiter gauged her interest in leading Epicrispr, which was then known as Epic Bio.By then, the startup had already started working on FSHD a disease that affected her husbands family, Salzman said. That, and the potential to use epigenetic editing against a wide range of diseases, convinced her to take the job.All of a sudden, I found a company that had addressed all the limitations I'd come across in genetic medicine, she said.Epicrispr raised a $55 million Series A round in 2022. Along with FSHD, its working on drugs for heterozygous familial hypercholesterolemia, alpha-1 antitrypsin deficiency, a pair of eye diseases and certain undisclosed blood cancers. All of its work is preclinical.The company was co-founded by Stanford researcher Stanley Qi, who worked closely with gene editing pioneer Jennifer Doudna at UC Berkeley. '

Genetic medicine startup Tune Therapeutics has raised another $175 million to develop therapies capable of tuning genes to treat disease, rather than editing them by cutting or replacing DNA directly.Tune disclosed the Series B round on Sunday, the eve of the J.P. Morgan Healthcare Conference, which regularly features as a curtain-raising meeting for the biotechnology and pharmaceutical sectors. It follows a busy week for private financing in biotech, adding to nearly $2 billion worth of investment announced by venture firms tracked by BioPharma Dive since Monday.Tunes lead therapy, dubbed Tune-401, is designed to treat chronic hepatitis B by silencing the viral DNA thats both integrated into an infected cells genome and circulating in loops within the cell. More than 250 million people worldwide are estimated to have chronic hepatitis B infections, which can cause liver failure and cancer. Researchers see altering gene expression via epigenetic editing as a way to block cells from producing more of the virus, rather than fighting the virus directly, as existing drugs do.Its thinking about addressing a leak by trying to shut off the faucet, said Akira Matsuno, one of Tunes co-founders and now its chief financial officer. Many therapies ... either try to slow down the pace or are really good at getting the water out of the tub, if you will. But ultimately, you have to be able to shut off the faucet.The company has begun a Phase 1 trial of Tune-401 in New Zealand and Hong Kong.Scientists have described epigenetic editing as more efficient and less disruptive than DNA-cutting technologies like CRISPR, proposing that it could also be used to turn genes on or off.We have these targeted, really specific therapies that do not confer DNA damage, Matsuno said. It opens up a lens with regards to the types of opportunities and settings we can go into over time.Tunes science builds on research by genetic medicine pioneers Charles Gersbach at Duke University and Fyodor Urnov at the University of California, Berkeley.The companys Series B round was co-led by New Enterprise Associates, Yosemite, Regeneron Ventures and Hevolution Foundation. When Tune launched in 2021, it raised a $40 million Series A.Chronic diseases of aging are accelerating in incidence, prevalence, and severity, and current approaches are simply inadequate, William Greene, chief investment officer at Hevolution Foundation, said in a statement. It is our belief that epigenetic editing may prove to be the transformative modality we need to enable a new era of regenerative medicine.Tune is one of several companies working to advance epigenetic editing as a drugmaking technology. Among its competitors in the field is nChroma Bio, which formed late last year from a merger between Chroma Bio and Nvelop Therapeutics. NChroma is also researching a potential treatment for hepatitis B, but has not entered human testing. Epicrispr Therapeutics, formerly known as Epic Bio, is researching treatments for several conditions including a form of muscular dystrophy and alpha-1 antitrypsin deficiency.Beyond epigenetic editing specifically, startups working in genetic medicine are seeing less investment flow into their field. Funding of cell and gene therapy developers declined in 2024 compared to the two years prior, according to BioPharma Dive data. '