- Preclinical data showcase scalable AAV manufacturing, compact gene activators, and expanded Cas effector tools for precise epigenetic modulation
SAN FRANCISCO--(BUSINESS WIRE)--#ASGCT25--Epicrispr Biotechnologies, a biotechnology company focused on developing curative therapies, today announced five presentations at the upcoming American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting, being held May 15-17, 2025, in New Orleans, Louisiana. The presentations will highlight the company’s progress across key areas, including its lead epigenetic therapeutic candidate EPI-321, novel Cas effectors for epigenome engineering, and innovations in compact modular epigenetic activators.
The accepted abstracts spotlight Epicrispr’s approach to modulating gene expression without altering the DNA sequence, enabling safer, tunable, and potentially reversible therapies. Two posters focus on the development and manufacturing of EPI-321, an rAAVrh74-delivered epigenetic therapy that will be initiating a first-in-human study for FSHD, a neuromuscular disease.
Oral Presentation:
Non-Human Primate (NHP) Safety Study of High-Dose EPI-321: A Novel AAV-Delivered Epigenetic Editing Gene Therapy for the Treatment of FSHD
- Presenter: Sid Boregowda, DVM, MS, Ph.D, DABT
- Session Title: Viral Vectors in Large Animal Models
- Date/Time: Wednesday, May 14, 2025, 3:45 – 5:30 PM CT
- Abstract Presentation Time: 4:45 – 5:00 PM CT
- Location: NOLA Theater B
Poster Presentation Details:
Directed Evolution and Characterization of Cas Effectors in Mammalian Cells for Expanded Epigenome Editing Space
- Presenter: Courtney Klappenbach, Senior Research Associate
- Poster Number: 617
- Date/Time: Tuesday, May 13, 2025, 6:00 – 7:30 PM CT
- Location: Poster Hall I2, New Orleans Ernest N. Morial Convention Center
Small Scale AAV Bioreactor Optimization Demonstrates Iterative Titer Gains of rAAVrh74 Serotype EPI-321, a CRISPR-mediated Epigenetic Therapy
- Presenter: James Kim, Associate Scientist
- Poster Number: 967
- Date/Time: Tuesday, May 13, 2025, 6:00 – 7:30 PM CT
- Location: Poster Hall I2, New Orleans Ernest N. Morial Convention Center
Compact DNA Demethylase-Activator Combination Modulators for CRISPR-mediated Epigenetic Gene Activation
- Presenter: Dan Hart, Ph.D, Head of Technology Development
- Poster Number: 1110
- Date/Time: Wednesday, May 14, 2025, 5:30 – 7:00 PM CT
- Location: Poster Hall I2, New Orleans Ernest N. Morial Convention Center
EPI-321 Development: Strategies to Establish a Scalable and Robust rAAVrh74 Upstream Manufacturing Process from 0.5 L to 1000 L Scale
- Presenter: Surabhi Godbole, Scientist I
- Poster Number: 1939
- Date/Time: Thursday, May 15, 2025, 5:30 – 7:00 PM CT
- Location: Poster Hall I2, New Orleans Ernest N. Morial Convention Center
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 www.epicrispr.com or follow us on LinkedIn.
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