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Epicrispr Biotechnologies
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Epigenetic gene-expression therapy platform
Epicrispr Biotechnologies
Work Here?
Epigenetic gene-expression therapy platform
Overview
Epicrispr Biotechnologies develops gene expression modulation therapies using epigenetics to treat complex diseases. Its Gene Expression Modulation System (GEMS) is a platform that lets researchers mix and match a toolbox of epigenomic modulators to tailor therapies by regulating gene expression in mammalian cells. Unlike competitors that rely on a single product, GEMS offers a flexible, customizable approach to create therapies for different diseases and patient needs. The company's goal is to expand genetic medicine by delivering targeted, epigenetically guided therapies through hospitals and clinics, while advancing scientific research.
About Epicrispr Biotechnologies
Industries
Enterprise Software
Biotechnology
Healthcare
Company Size
11-50
Company Stage
Series C
Total Funding
$213M
Headquarters
South San Francisco, California
Founded
2018
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Simplify's Take
What believers are saying
- August 2026 Series C raised $90 million for pivotal EPI-321 studies.
- EPI-321 completed Phase 1/2 enrollment, with additional data expected later in 2026.
- May 2026 Forge partnership secures cGMP AAV manufacturing for U.S., New Zealand, Australia.
What critics are saying
- EPI-321 remains single-asset; any safety or efficacy miss strands Epicrispr’s platform.
- FSHD is crowded; Avidity, Dyne, and others chase disease-modifying muscle therapies.
- Pivotal trial execution and AAV manufacturing scale-up now determine 2027 financing access.
What makes Epicrispr Biotechnologies unique
- Epicrispr’s GEMS platform modulates gene expression without cutting DNA, unlike classic CRISPR.
- EPI-321 targets DUX4 silencing in FSHD, a one-time AAV-delivered approach.
- August 2026 Series C drew Octagon, Janus Henderson, Fidelity, and Sanofi Ventures.
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Funding
Total Funding
$213M
Above
Industry Average
Funded Over
3 Rounds
Industry standards
Benefits
Health Insurance
Paid Vacation
Flexible Work Hours
Remote Work Options
401(k) Retirement Plan
Gym Membership
Mental Health Support
Wellness Program
Conference Attendance Budget
Professional Development Budget
Training Programs
Employee Stock Purchase Plan
Stock Options
Company Equity
Parental Leave
Family Planning Benefits
Fertility Treatment Support
Growth & Insights and Company News
6 month growth
↓ -4%1 year growth
↓ -2%2 year growth
↓ -6%Epicrispr Biotechnologies Closes $90 Million Series C Financing to Advance First-in-Class Epigenetic Therapy Toward Pivotal Studies in FSHD
Epicrispr's $90m round bets big on epigenetics. Oversubscribed Series C pushes EPI-321 toward pivotal trials, testing whether "silencing genes without editing them" can become real therapy. Epicrispr Biotechnologies has closed a $90 million Series C financing round, and the syndicate behind it reads like a checklist of biotech credibility: Octagon Capital and Janus Henderson Investors co-led, joined by Fidelity Management & Research Company, Cormorant Asset Management, Duquesne Family Office, Sanofi Ventures, funds managed by abrdn Inc., Angelini Ventures, Readout Capital, and a run of existing backers who came back for more. For a clinical-stage company with a single lead asset still in first-in-human testing, an oversubscribed round of this size is a meaningful vote of confidence and a signal that investors are increasingly willing to bet on epigenetics as a therapeutic modality in its own right, not just an academic curiosity sitting beside gene editing. The proceeds have a clear job: push EPI-321, Epicrispr's lead candidate for facioscapulohumeral muscular dystrophy (FSHD), into pivotal clinical development, while also accelerating the broader pipeline of programmable epigenetic medicines built on the company's Gene Expression Modulation System, or GEMS platform. That's a three-part mandate - fund a pivotal trial, expand a pipeline, and scale up manufacturing capability - that's typical of a company trying to move from "promising early data" to "commercially credible therapeutic company" in one financing cycle. FSHD is a good test case for that ambition. It's a progressive, currently untreatable form of muscular dystrophy driven by inappropriate activation of the DUX4 gene in muscle tissue, and there's no approved disease-modifying therapy on the market. A company that can durably silence DUX4 without altering the underlying DNA sequence would be addressing a genuine unmet need rather than competing in an already-crowded therapeutic category. Why 'epigenetic' is the operative word. The distinction Epicrispr is drawing matters more than it might first appear. Gene editing technologies change the DNA sequence itself, a permanent, one-way intervention. Epigenetic modulation, by contrast, changes how genes are expressed without touching the sequence underneath, in principle offering a more reversible and potentially more precisely tunable form of control. GEMS is built to selectively activate or silence disease-causing genes using that logic, and EPI-321 is its first attempt to prove the approach works in patients, not just in a dish or an animal model. The early clinical readout gives that thesis some real support. In its ongoing Phase 1/2 first-in-human trial, EPI-321 has shown a favorable safety profile alongside statistically significant increases in lean muscle volume, measured by MRI, along with biomarker changes consistent with DUX4 suppression, all following a single administration. That last detail is worth sitting with: this is designed as a one-time intravenous dose, delivered to muscle tissue via a clinically validated AAV vector, aiming for durable effect rather than a chronic dosing regimen. Enrollment in the trial is now complete, with additional data expected later this year, which is presumably the next real catalyst investors and clinicians alike will be watching for. The investor logic. CEO Amber Salzman framed the round as validation of the company's ability to translate a differentiated platform into clinical progress, and the investor commentary echoes that framing closely. Octagon Capital's Anran Li, who is joining Epicrispr's board as part of the deal, pointed to the speed with which the company moved from platform science to encouraging early clinical data as the reason for stepping in, a pace that matters in a financing environment where biotech investors have grown considerably more selective about which platform companies get funded through to pivotal-stage trials. That selectivity is exactly what makes this round notable. Series C rounds for clinical-stage biotechs with one asset in early trials don't reliably attract nine named institutional investors in an oversubscribed raise unless the underlying data is doing real work. The presence of strategic and specialist life-sciences investors alongside generalist asset managers suggests a read-through: epigenetic silencing is being treated less as a niche mechanism and more as a plausible platform technology, one capable of generating a pipeline rather than a single drug. What to watch next. The company is explicit that EPI-321 is only the leading edge of a broader ambition - additional programs are already advancing across multiple therapeutic areas built on the same GEMS platform. For a technology this early in its clinical validation, the real proof point isn't the financing itself but what comes out of the additional EPI-321 data expected later this year, and whether the biomarker and functional signals seen so far translate into a credible pivotal-trial design. If they do, Epicrispr's Series C may end up looking less like a single funding milestone and more like the moment programmable epigenetic medicine started being treated as an investable category in its own right.
Epicrispr Biotechnologies named to Fast Company's annual list of the World's Most Innovative Companies of 2026. SAN FRANCISCO-(BUSINESS WIRE)-#FCMostInnovative-Epicrispr Biotechnologies, a clinical-stage company pioneering gene-modulating therapies, is proud to have been named to Fast Company's prestigious list of the World's Most Innovative Companies of 2026, ranking among the top companies in the Biotech category. This year's list shines a spotlight on businesses that are shaping industry and culture through their innovations. Alongside the World's 50 Most Innovative Companies, Fast Company recognizes 720 honorees across 59 sectors and regions. "We are honored to be recognized by Fast Company as one of the world's most innovative companies," said Amber Salzman, Ph.D., Chief Executive Officer, Epicrispr Biotechnologies. "At Epicrispr, our focus is on unlocking the full potential of gene regulation to treat diseases that have long been considered untreatable. This recognition reflects the strength of our platform and the urgency of delivering transformative therapies to patients with neuromuscular diseases such as FSHD." FSHD, or facioscapulohumeral muscular dystrophy, is a progressive genetic muscle disorder that affects approximately 1 in 8,000 individuals worldwide, leading to skeletal muscle degeneration and severe loss of function. There are currently no approved disease-modifying therapies for FSHD. Epicrispr's lead candidate, EPI-321, is the first and only epigenetic editing therapy to receive IND authorization within the U.S. and is currently being evaluated in a first-in-human Phase 1/2 trial in the U.S., New Zealand, and Australia. EPI-321 has received FDA Fast Track Designation, Orphan Drug Designation, and Rare Pediatric Disease Designation for FSHD. Designed to suppress pathogenic gene activity without cutting DNA, EPI-321 targets the root cause of FSHD and has the potential to be a one-time, disease-modifying, curative therapy for this progressive neuromuscular condition affecting more than one million people globally. To date, eight participants have been dosed, with early data showing favorable improvements across surrogate biomarkers and functional measures, with no serious adverse events reported. While clinical data is preliminary, there is compelling evidence of patients gaining muscle strength, increasing speed in various timed assessments, and gains in muscle mass. The World's Most Innovative Companies is Fast Company's trademark franchise and one of its most anticipated editorial efforts of the year. To determine honorees, Fast Company's editors and writers review companies driving progress around the world and across industries, evaluating thousands of submissions through a competitive application process. The result is a globe-spanning guide to innovation today, from early-stage startups to some of the most valuable companies in the world. "Our list of the Most Innovative Companies is about spotlighting organizations that don't just adapt to change - they drive it," said Brendan Vaughan, editor-in-chief of Fast Company. "The companies we honor this year are redefining what leadership looks like in 2026, pairing bold ideas with measurable impact and turning breakthrough innovation into real-world value. They are setting the pace for their industries and offering a blueprint for what sustained innovation can achieve." The full list of Fast Company's Most Innovative Companies honorees can now be found at fastcompany.com. It will also be available on newsstands beginning March 31, 2026. 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 and 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. Learn more at www.epicrispr.com or follow us on LinkedIn. Editor details. * Company: * Businesswire Last Updated: 24-Mar-2026
Epicrispr Biotechnologies to present at 44th Annual J.P. Morgan Healthcare Conference. SAN FRANCISCO-(BUSINESS WIRE)-Epicrispr Biotechnologies, a clinical-stage company pioneering gene-modulating therapies, today announced that Amber Salzman, Ph.D., Chief Executive Officer, will present at the 44th Annual J.P. Morgan Healthcare Conference on Thursday, January 15, 2026, at 9:00 a.m. PT. "Our first year as a clinical-stage company has reinforced the promise of epigenetic editing to precisely and durably control gene expression without cutting DNA," said Amber Salzman, Ph.D., CEO, Epicrispr. "We look forward to sharing our progress at the J.P. Morgan Healthcare Conference as we begin generating meaningful patient data and advance a pipeline of one-time, gene-modulating therapies for difficult-to-treat diseases, beginning with neuromuscular disease." A live webcast of the presentation will be available on the Epicrispr website under the News section at: https://epicrispr.com/news. An archived replay of the webcast will remain available for approximately 30 days following the event. 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. Learn more at www.epicrispr.com or follow us on LinkedIn.
RhyGaze raised $86 million (series A, Jan) for its cone optogenetics vision-restoration platform, while Epicrispr secured $68 million (series B, Mar) to advance EPI-321, the first epigenetic therapy in trials for FSHD.
Epicrispr Biotechnologies is Hiring for 5 Jobs on Simplify!
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About Epicrispr Biotechnologies
Industries
Enterprise Software
Biotechnology
Healthcare
Company Size
11-50
Company Stage
Series C
Total Funding
$213M
Headquarters
South San Francisco, California
Founded
2018
Epicrispr Biotechnologies is Hiring for 5 Jobs on Simplify!
Find jobs on Simplify and start your career today