Simultaneous epigenetic editing of three genes in primary human T cells resulted in no indels or genomic alterations, while simultaneous triple editing with a Cas9 nuclease generated significant chromosomal abnormalities

Boston, MA, May 19, 2023 – Chroma Medicine, Inc., (Chroma) a genomic medicine company pioneering single-dose epigenetic editing therapeutics, today presented data demonstrating the advantages of epigenetic editing for multiplex gene regulation at the American Society of Gene & Cell Therapy (ASGCT) 26^th Annual Meeting in Los Angeles.

Chroma’s epigenetic editing platform harnesses the cell’s endogenous mechanism for regulating gene expression to durably modulate transcription without cutting or nicking the DNA, offering a potentially safer approach for multiplex editing. This avoids challenges associated with gene editing methods that rely upon DNA breaks to alter gene expression, which have the potential to induce unnecessary risks, including chromosomal rearrangements when multiplex editing several genes simultaneously.

Data presented today at ASGCT demonstrate the key advantages of epigenetic editing for multiplex gene regulation compared to Cas9-based approaches. Application of Chroma’s epigenetic editor to three different genes simultaneously accomplished durable silencing in primary human T cells. Sequencing and single-cell imaging assays indicated that multiplex epigenetic editing did not induce indels, chromosomal rearrangements, or other unintended genomic alterations compared to controls. Conversely, cells simultaneously edited using a Cas9 nuclease exhibited a significant number of translocations and other inadvertent breaches to genomic integrity.

“These results underscore one of the key advantages of harnessing nature’s innate mechanism for precise gene regulation. By modulating expression without cutting DNA, we can safely and efficiently target multiple genes simultaneously,” said Vic Myer, Ph.D., Chroma’s President and CSO. “Taken together with results presented yesterday demonstrating durable and highly efficient silencing of PCSK9 and hepatitis B virus, these data validate our platform and its distinct advantages, positioning us to advance a broader pipeline of epigenetic editing therapeutics.”

“Data presented at ASGCT by Chroma and our scientific founders represent an important step toward rapidly advancing these medicines to patients,” said Catherine Stehman-Breen, M.D., M.S., Chroma’s CEO. “We are committed to rigorously progressing our pioneering epigenetic editing technology and programs and are pleased to present data supporting the therapeutic promise of epigenetic editing for gene regulation while preserving genomic integrity.”

Details for Chroma’s upcoming oral presentation at ASGCT are as follows:

Title: Multiplexed Editing Without Chromosomal Rearrangements Using Epigenetic Editors
Presenter: Sahar Abubucker, Senior Director, Data Science
Date and Time: Friday, May 19^th, 4:45p PT
Session: Genome & Epigenome Editing Technologies

Chroma’s presentations will be available on the company website following the meeting.

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About Chroma Medicine

Chroma Medicine is a biotechnology company pioneering a new class of genomic medicines that harness epigenetics, nature’s innate mechanism for gene regulation, to deliver precise, programable single-dose therapeutics while preserving genomic integrity. The company was founded by the world’s foremost experts in genomic research and is led by a veteran team of industry leaders and scientists with deep experience in genomic medicine, drug discovery, and development. For more information, please visit chromamedicine.com or follow the company on LinkedIn and Twitter.

Media Contact

Lia Dangelico
Verge Scientific Communications
540.303.0180
ldangelico@vergescientific.com