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In vivo data provide compelling proof-of-concept for Chroma's epigenetic editing platform to enable highly efficient, specific, and durable gene regulation

Data demonstrate the potential of the company's epigenetic editors for multiplex editing while preserving genomic integrity

BOSTON, May 2, 2023 /PRNewswire/ -- Chroma Medicine, Inc., (Chroma) a genomic medicine company pioneering single-dose epigenetic editing therapeutics, today announced two oral data presentations that showcase the potential of its epigenetic editing platform at the American Society of Gene & Cell Therapy (ASGCT) 26^th Annual Meeting, held May 16-20, 2023 in Los Angeles, California. The presentations include the first in vivo proof-of-concept data for Chroma's epigenetic editing platform along with data demonstrating the key advantages of epigenetic editing for multiplex gene regulation. Chroma plans to release data from additional pipeline programs at upcoming scientific conferences.  

"These data showcase the power of harnessing an innate system for gene regulation," said Vic Myer, Ph.D., Chroma's President and CSO. "They also demonstrate the vast potential of this approach to unlock a new generation of genomic medicines and enable Chroma's broader pipeline of epigenetic editing therapeutics."

The presentations will be archived on the Chroma website following the meeting.

Details and overviews for the presentations are as follows:

Title: Development of a Human PCSK9-Targeted Epigenetic Editor with Durable, Near-Complete In Vivo Silencing Efficiency
Presenter: Aron Jaffe, Ph.D., Senior Vice President, Head of Research
Date and Time: Thursday, May 18^th, 2:30p PT
Session: Gene Targeting and Gene Correction: Liver
Summary: 

• Epigenetic editors have been shown to durably suppress gene expression by making localized changes in DNA methylation without cutting, nicking, or altering the DNA sequence.
• The work presented at ASGCT describes the development of Chroma's epigenetic editor targeting human PCSK9 (hPCSK9), a well-established genetic target for addressing hypercholesterolemia which is characterized by elevations in low-density lipoprotein (LDL) cholesterol.
• Preclinical studies in a transgenic mouse model indicate that Chroma's optimized epigenetic editor can achieve near-complete silencing of hPCSK9 with high efficiency, durability, and specificity.

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
Summary: 

• The ability to multiplex edit, or simultaneously alter the expression of multiple genes, is desirable for many applications, including for cell-based therapeutics.
• Genome editing approaches that rely on double-stranded DNA breaks to modulate gene expression have potential for undesirable effects, including large deletions and translocations. This potential is amplified with multiplex editing of several target genes.
• Data presented at ASGCT demonstrate the ability of epigenetic editors to facilitate genotoxicity-free multiplexed editing in healthy donor-derived T cells, as compared to Cas9-mediated editing.

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.

SOURCE Chroma Medicine