Latest news with #Epicrispr
Business Wire
12-05-2025
- Business
- Business Wire
Epicrispr Biotechnologies Named Finalist in XPRIZE Healthspan Competition FSHD Bonus Prize
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Epicrispr Biotechnologies, a biotechnology company focused on developing curative therapies, today announced it has been named a Finalist Team in XPRIZE Healthspan FSHD Bonus Prize, earning a $250,000 Milestone Award for its work addressing facioscapulohumeral muscular dystrophy (FSHD), one of the most prevalent forms of muscular dystrophy globally. Epicrispr was one of eight global teams selected from hundreds of applicants to receive the $10M Milestone 1 FSHD Bonus Prize, part of the broader $101M XPRIZE Healthspan initiative supported by Hevolution Foundation and SOLVE FSHD. The award recognizes groundbreaking innovation aimed at extending healthy human lifespan and improving outcomes for patients affected by degenerative diseases of aging. 'Being recognized by XPRIZE as a finalist and Milestone 1 recipient of the FSHD Bonus Award is a major validation of our platform and mission,' said Amber Salzman, Ph.D., CEO, Epicrispr Biotechnologies. 'Our team is deeply motivated by the urgent need for novel therapies that can meaningfully help patients with FSHD and other epigenetically driven diseases.' 'We designed the XPRIZE Healthspan FSHD Bonus Prize to spark innovative solutions for FSHD that could have ripple effects across aging, muscle regeneration, and healthspan,' said Jamie Justice, Ph.D., Executive Director of XPRIZE Healthspan. 'Each of the eight finalist teams receiving this milestone award are demonstrating compelling approaches to address FSHD and push the boundaries of what aging with FSHD can look like.' Epicrispr's approach leverages proprietary technology to modulate gene expression without cutting DNA, offering a new class of therapeutics with the potential to precisely control disease-causing genes. Its lead program targets DUX4, the gene at the root of FSHD, with a goal of restoring muscle function and halting disease progression through safe, tunable interventions. Finalists were selected by a panel of independent judges based on the scientific rigor, feasibility, and transformative potential of their work. Epicrispr will be honored at the XPRIZE Healthspan Awards Ceremony and Investor Summit in New York City, taking place May 12–14, 2025. About Epicrispr Biotechnologies Epicrispr Biotechnologies is a biotechnology company pioneering gene-modulating therapies, leading with treatments for neuromuscular diseases. The company's proprietary Gene Expression Modulation System (GEMS) enables precise, durable control of gene expression, unlocking first-in-class treatments for previously untreatable conditions. Epicrispr's lead program, EPI-321 is in clinical trials for FSHD, and the company is advancing additional gene-modulating therapies. Epicrispr also has a research collaboration with Kite Pharma to develop next-generation CAR T-cell therapies. Learn more at or follow us on LinkedIn. About XPRIZE XPRIZE is the recognized global leader in designing and executing large-scale competitions to solve humanity's greatest challenges. For over 30 years, our unique model has democratized crowd-sourced innovation and scientifically scalable solutions that accelerate a more equitable and abundant future. Donate, learn more, and co-architect a world of abundance with us at
Yahoo
26-03-2025
- Health
- Yahoo
Epicrispr banks $68M to test epigenetic editing on rare muscle disease
This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. 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 startup's 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 company's Wednesday statement. The San Francisco Bay Area biotech's Series B round was led by Ally Bridge Group and involved Solve FSHD, an advocacy group formed by Lululemon Athletica founder Chip Wilson. 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, it's 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 company's 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.' Salzman 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 Salzman's 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 husband's 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, it's 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.
