PRESS RELEASES

Boston, December 10, 2023 – Chroma Medicine, Inc., (Chroma) a genomic medicine company pioneering single-dose epigenetic editing therapeutics, today presented data demonstrating the potential of its multiplex epigenetic editing platform to enhance functional allogeneic chimeric antigen receptor (CAR) T cells at the American Society of Hematology (ASH) 65th Annual Meeting in San Diego.

Allogeneic CAR T cell products offer advantages over autologous CAR T cells and have the potential to broaden access to cancer immunotherapy; however, their clinical effect remains limited by lack of durable response and challenges related to immunogenicity and graft-versus-host disease. Incorporating additional edits may help produce cells resistant to rejection—potentially improving allogeneic T cell persistence—but using editing technologies that rely on double- or single-stranded DNA breaks can result in unintended genomic changes. Chroma’s epigenetic editing technology has the potential to provide durable modulation of gene expression without cutting or nicking the DNA, making it well-suited for simultaneous targeting of multiple genes to enhance generation of persistent allogeneic CAR T cells.

“The data presented at ASH illustrate the tremendous potential of epigenetic editing to accelerate allogeneic CAR T approaches by enhancing functional allogeneic cells to avoid common genotoxic challenges observed in nuclease-based gene editing and base editing,” said Vic Myer, Ph.D., Chroma’s President and CSO. “We believe harnessing nature’s innate mechanism for gene regulation unlocks vast opportunity to efficiently, effectively, and simultaneously silence many target genes, and the data presented underscore the strength of this approach in ex vivo cell therapies.”

The poster presentation shows Chroma’s epigenetic editor in primary T cells induces durable silencing of T cell receptor and MHC class I and MHC class II expression, maintained through restimulation. The data also demonstrate that multiplex epigenetic editing of CAR T cells does not interfere with cytotoxic anti-tumor function, further highlighting the potential to create CAR T cell therapies with greater accessibility, durability, and efficacy for patients.

Details for Chroma’s poster presentation at the 2023 ASH Annual Meeting are as follows:

Title: Durable Multiplex Epigenetic Editing for Generation of Allogeneic CAR T Without Chromosomal Rearrangements
Presenter: Jamie Schafer, Ph.D., Associate Director, Ex Vivo Program Sciences, Chroma Medicine
Session: 703. Cellular Immunotherapies: Basic and Translational: Poster II
Session Date and Time: Sunday, December 10, 2023, 6:00 – 8:00p PT

The poster presentation is available on Chroma’s website.

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About Chroma Medicine

Chroma Medicine is a biotechnology company pioneering a new class of genomic medicines that harnesses epigenetics, nature’s innate mechanism for gene regulation, to deliver precise, programmable single-dose therapeutics while preserving genomic integrity. The company’s modular platform enables development of medicines that can address a wide range of complex diseases, whether they require silencing, activation, or targeting multiple genes at once. Chroma 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 X (formerly known as Twitter).

Media Contact

Lia Dangelico

Verge Scientific Communications

540.303.0180

lia.dangelico@vergescientific.com

Boston, November 11, 2023Chroma Medicine, Inc., (Chroma) a genomic medicine company pioneering single-dose epigenetic editing therapeutics, today presented new preclinical data demonstrating that a single administration of its epigenetic editor efficiently and durably lowered PCSK9 and cholesterol levels in non-human primates in a late-breaking oral presentation at the 2023 American Heart Association (AHA) Scientific Sessions, held November 11-13 in Philadelphia.

The data presented provide strong evidence for the potential of Chroma’s epigenetic editing platform to silence expression of PCSK9 in the liver and to induce a durable reduction in low-density lipoprotein-cholesterol (LDL-C) levels, a key factor for reducing risk of atherosclerotic cardiovascular disease (ASCVD), without cutting or nicking the DNA.

The PCSK9 gene actively promotes the degradation of LDL receptors, thereby reducing the ability of the liver to clear cholesterol from the blood. Genetic and pharmacologic reductions in PCSK9 levels have been shown to correlate with decreased cardiovascular events, making it an established target for prevention of ASCVD.

“We believe our epigenetic editing approach offers the potential for highly efficacious, specific, and durable silencing of PCSK9 in the liver while preserving genomic integrity and avoiding the inherent risks of editing approaches that cut or nick the DNA,” said Vic Myer, Ph.D., Chroma’s President and Chief Scientific Officer. “These results not only validate the power of our platform to effectively silence PCSK9 but also provide strong evidence of the resulting impact on key therapeutic biomarkers.”

The work details Chroma’s human PCSK9-targeting epigenetic editor, which was shown to efficiently and durably reduce PCSK9 levels in vivo through 10 months of follow up after a single administration in human transgenic mice. The data also show that the silencing was maintained in mice pre- and post- partial hepatectomy, the standard surgical model for induction of liver regeneration. In non-human primates, a significant reduction in circulating PCSK9 (80%) levels was achieved, resulting in a 58% reduction in LDL-C levels.

These studies also show a clearer link between Chroma’s PCSK9 epigenetic editor’s molecular action of CpG methylation and PCSK9 silencing. Application of the company’s epigenetic editor was found to be highly correlated with targeted CpG methylation at the PSCK9 locus in vivo and durable through tissue regeneration in the partial hepatectomy model.

“Developing single-dose therapies is a crucial step for disrupting the current treatment paradigm for lowering LDL-cholesterol and reducing the risk of serious cardiovascular disease,” said Catherine Stehman-Breen, M.D., Chroma’s Chief Executive Officer. “Data presented today signal the potential of our epigenetic editing platform to deliver precise and durable gene regulation by leveraging the cell’s natural mechanism, bringing us another step closer to delivering better therapies to patients.”

Details for Chroma’s presentation at the 2023 AHA Scientific Sessions are as follows:

Title: A Single Administration of an Epigenetic Editor Targeting Human PCSK9 Robustly and Durably Lowers Cholesterol In Vivo
Presenter: Frederic Tremblay, Ph.D., Vice President, Head of In Vivo Programs, Chroma Medicine
Date and Time: Saturday, November 11, 2023, 9:45 – 9:55a ET
Session: Late Breaking Basic Science Abstract Oral Session

The presentation will be available on the Chroma website following the meeting.

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About ASCVD

Chronically elevated blood levels of low-density lipoprotein-cholesterol (LDL-C) can lead to increased risk for early onset atherosclerotic cardiovascular disease (ASCVD), heart attack, and stroke. ASCVD is the leading cause of death in the United States and globally. Current therapeutic interventions often fail to lower LDL-C below designated therapeutic thresholds and require frequent dosing, leading to treatment adherence challenges. The PCSK9 gene actively promotes the degradation of LDL receptors, which are responsible for clearing LDL-C from the blood, thereby reducing the ability of the liver to clear cholesterol from the blood. Genetic and pharmacologic reductions in PCSK9 have been associated with decreased LDL-C levels and reduced risk for cardiovascular events. Chroma is advancing an in vivo epigenetic editing therapeutic designed to effectively and durably silence the PCSK9 gene without cutting or nicking the DNA, resulting in decreased PCSK9 protein levels and leading to lower LDL-C levels and reduced risk of ASCVD.

About Chroma Medicine

Chroma Medicine is a biotechnology company pioneering a new class of genomic medicines that harnesses epigenetics, nature’s innate mechanism for gene regulation, to deliver precise, programmable single-dose therapeutics while preserving genomic integrity. The company’s modular platform is designed to enable the development of medicines that address a wide range of complex diseases, whether they require silencing, activation, or targeting multiple genes at once. Chroma was founded by 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 X (formerly known as Twitter).

Media Contact

Lia Dangelico

Verge Scientific Communications

540.303.0180


Boston, MA, November 2, 2023 – Chroma Medicine, Inc., (Chroma) a genomic medicine company pioneering single-dose epigenetic editing therapeutics, today announced that it will present a poster demonstrating the potential of its multiplex epigenetic editing platform at the American Society of Hematology (ASH) 65th Annual Meeting, taking place December 9-12, 2023, in San Diego, CA.

The ASH abstract contains data showing the potential of multiplex epigenetic editing to produce functional allogeneic CAR T cells that do not exhibit a graft-versus-host-disease (GVHD) response, resist CD8+ and CD4+ T cell alloresponses, and resist the NK missing-self response normally consequent of reduced MHC class I expression, without introduction of genotoxic events.

Details of the poster presentation are:

Title: Durable Multiplex Epigenetic Editing for Generation of Allogeneic CAR T without Chromosomal Rearrangements
Presenter: Jamie Schafer, Ph.D., Associate Director, Ex Vivo Program Sciences, Chroma Medicine
Session: 703. Cellular Immunotherapies: Basic and Translational: Poster II
Session Date and Time: Sunday, December 10, 2023, 6:00-8:00 PM

The full abstract is available through the ASH conference website.

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About Chroma Medicine

Chroma Medicine is a biotechnology company pioneering a new class of genomic medicines that harnesses epigenetics, nature’s innate mechanism for gene regulation, to deliver precise, programmable single-dose therapeutics while preserving genomic integrity. The company’s modular platform enables development of medicines that can address a wide range of complex diseases, whether they require silencing, activation, or targeting multiple genes at once. Chroma 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 X (formerly known as Twitter).

Media Contact

Lia Dangelico

Verge Scientific Communications

540.303.0180

ldangelico@vergescientific.com

Boston, MA, September 27, 2023Chroma Medicine, Inc., (Chroma) a genomic medicine company pioneering single-dose epigenetic editing therapeutics, today announced the addition of innovative biotech business leader Michael A. Kelly to its Board of Directors.

“Michael brings decades of strategic finance and business operations experience to Chroma and builds on the deep expertise of our current board and leadership team. We are excited to welcome him as we pursue the promise of epigenetic editing for patients,” said Catherine Stehman-Breen, M.D., Chief Executive Officer of Chroma.

Mr. Kelly is the Founder and President of Sentry Hill Partners, LLC, a global life sciences transformation and management consulting firm that he founded in 2018. In addition to Chroma, he serves as an independent member on the boards of Prime Medicine, Amicus Therapeutics, DMC Global Inc., and NeoGenomics Laboratories. Previously, he was a senior executive at Amgen, where he most recently served as Senior Vice President, Global Business Services. Mr. Kelly also twice served as Amgen’s acting Chief Financial Officer as well as the Vice President & Chief Financial Officer for International Commercial Operations. He has held senior positions at Tanox, Biogen, and Nutrasweet Kelco Company, a division of Monsanto Life Sciences. He serves on the Council of Advisors and was the former audit committee chair for Direct Relief, a humanitarian aid organization focused on health outcomes and disaster relief. Kelly obtained his B.Sc. in Business Administration with a concentration in Finance and Industrial Relations from Florida A&M University. 

“Genomic medicine is a rapidly growing, cutting-edge field and Chroma’s highly specific and durable epigenetic editors have great potential to be transformative in the treatment of serious diseases,” said Mr. Kelly. “I am thrilled to be part of this important mission as the company continues to build the leading platform of epigenetic editing therapies.”

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, programmable 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 strong 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 us on LinkedIn and X (formerly known as Twitter).

Media Contact

Lia Dangelico

Verge Scientific Communications

540.303.0180

lia.dangelico@vergescientific.com

Boston, MA., September 21, 2023 — Chroma Medicine, Inc. (Chroma), a genomic medicine company pioneering single-dose epigenetic editing therapeutics, today announced it was named to the Endpoints 11 2023 list of the most promising biotechs by Endpoints News. This annual award celebrates companies with leading-edge science that are pioneering the development of new medicines.

Building on groundbreaking research from the world’s foremost genomic medicine experts, Chroma is rapidly advancing a new class of therapeutics that harness epigenetics, nature’s innate mechanism for gene regulation, to achieve unparalleled control of gene expression without cutting, nicking, or altering the DNA sequence. After securing a $135 million Series B financing in early 2023, the company presented compelling in vivo data at the 2023 ASGCT Annual Meeting in Los Angeles showing 99% silencing of two distinct gene targets, demonstrating the promise of its epigenetic editing platform.

“We are honored to be acknowledged alongside our peer companies where together we are breaking new ground,” said Catherine Stehman-Breen, M.D., Chief Executive Officer of Chroma. “I am deeply grateful to our founders, investors, and partners for their ongoing support and to our Chroma team of dedicated and passionate individuals diligently working to progress our portfolio of highly differentiated epigenetic editing therapeutics to bring hope to patients living with serious diseases.”

Honorees were announced and recognized in an event held yesterday at the State Room in Boston. See the full list from Endpoints News.

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, programmable 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 us on LinkedIn and X (formerly known as Twitter).

Media Contact

Lia Dangelico

Verge Scientific Communications

540.303.0180

lia.dangelico@vergescientific.com

Boston, MA, September 20, 2023Chroma Medicine, Inc., (Chroma) a genomic medicine company pioneering single-dose epigenetic editing therapeutics, today presented positive preclinical data demonstrating the potential of its epigenetic editors to produce efficacious, specific, and durable silencing of the hepatitis B virus (HBV) during the HBV International Meeting 2023, held September 19-23 in Kobe, Japan.

Chronic hepatitis B is incurable, increases the risk of developing liver cirrhosis and hepatocellular cancer, and impacts nearly 300 million people worldwide, accounting for more than 800,000 deaths per year. Antiviral therapy can suppress viral replication, but requires lifelong dosing and few patients achieve functional cure due to the persistence of covalently closed circular DNA (cccDNA) and the integration of HBV DNA into the host genome.

Current gene editing approaches rely on targeted DNA breaks, which introduce risk for translocations or increased viral integration into the host genome, limiting their utility for effectively addressing HBV. By harnessing the endogenous cellular mechanism for regulating gene expression, Chroma’s epigenetic editors provide highly efficacious, specific, and durable silencing of HBV expression with the potential for a functional cure without introducing the unintended chromosomal effects associated with traditional gene editing approaches.

Chroma identified several epigenetic repressors across the HBV genome, targeting HBV cccDNA and integrated DNA from multiple genotypes, that durably and efficiently reduced hepatitis B surface antigen (HBsAg) and hepatitis B e antigen (HBeAg) in cellular models of HBV infection.

The data show a single administration of Chroma’s epigenetic editor robustly and durably repressed serum viral markers in transgenic HBV mice, with 5 of 6 (83%) animals exhibiting undetectable HBV DNA and HBsAg at the last time point tested (> 5 months).

“Our epigenetic editing approach holds the potential for highly efficacious, specific, and durable silencing of both episomal and integrated HBV, independent of virus genotype,” said Vic Myer, Ph.D., Chroma’s President and CSO. “These results are an encouraging demonstration of our platform potential as we advance a new class of genomic medicines toward the clinic and bring hope for functional cures to patients living with chronic HBV.”

Details for Chroma’s presentation at the HBV International Meeting are as follows:

Title: Development of HBV-targeting epigenetic repressors with deep, durable in vivo silencing of viral markers
Presenter: Yesseinia Anglero-Rodriguez, Ph.D., Principal Scientist, Chroma Medicine
Date and Time: Wednesday, September 20, 2023, 5:15p – 7:15p JST (4:15a – 6:15a ET)
Session: Poster Session I

The presentation will be available on the Chroma 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, programmable, 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 us on LinkedIn and X (formerly known as Twitter).

Media Contact
Lia Dangelico
Verge Scientific Communications
540.303.0180
ldangelico@vergescientific.com

Brisbane, California, and Boston, Massachusetts, JULY 20, 2023 – Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a clinical-stage genomic medicine company and Chroma Medicine, Inc. (Chroma) a genomic medicine company pioneering single-dose epigenetic editing therapeutics, today announced they have entered into a research evaluation, option and license agreement to develop epigenetic medicines leveraging zinc finger proteins (ZFPs) for sequence-specific DNA recognition. Over the course of two decades, Sangamo has built and validated the world’s largest library of ZFPs, deploying them to address numerous therapeutically relevant targets.  Advancing the transformative potential of epigenetic editing, Chroma is expanding the versatility of its platform by leveraging Sangamo’s ZFPs. Following a research evaluation period, Chroma has the option to license the ZFPs for potential worldwide development and commercialization of epigenetic medicines for certain therapeutic targets.

“Sangamo is the world leader in zinc finger protein genomic engineering, and we are very proud of the range and depth of our capabilities in this area,” said Jason Fontenot, Ph.D., Chief Scientific Officer at Sangamo. “We believe our ability to rapidly design and engineer highly potent and specific ZFPs can provide unique and highly valuable capability beyond what is available with competing technologies. We are constantly seeking to deploy our technology with partners outside of our core neurology focus area and are very happy to explore combining our ZF technology with Chroma’s unique capabilities. We believe that this work will further validate the importance of zinc fingers as an ideal platform to support epigenetic editing.”

Under the terms of the agreement, Chroma will evaluate novel Sangamo ZFPs for specified collaboration targets outside of the central nervous system in exchange for an upfront technology access payment. If Chroma exercises its option for any or all targets, Sangamo would be eligible to receive an option exercise payment, in addition to potential development and commercial milestone payments, as well as royalties on any Chroma products incorporating the licensed ZFPs. Chroma will lead and fund all research, development, manufacture, and commercialization of products incorporating the licensed Sangamo ZFPs.  

“As a leader in epigenetic editing, Chroma has advanced and optimized our platform, showing highly efficient, specific, and durable gene silencing in vivo and the ability to accomplish multiplex epigenetic editing without induction of indels or chromosomal rearrangements,” said Vic Myer, Ph.D., President and Chief Scientific Officer of Chroma. “Leveraging Sangamo’s leading zinc finger protein engineering capabilities expands our platform optionality, further positioning Chroma to progress a broad portfolio of epigenetic editing therapeutics that spans several indications.”

About Sangamo Therapeutics 

Sangamo Therapeutics is a clinical-stage biopharmaceutical company with a robust genomic medicines pipeline. Using ground-breaking science, including our proprietary zinc finger genome engineering technology and manufacturing expertise, Sangamo aims to create new genomic medicines for patients suffering from diseases for which existing treatment options are inadequate or currently don’t exist. To learn more, visit www.sangamo.com and connect with us on LinkedIn

and Twitter.

About Chroma Medicine, Inc. 

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.

Sangamo Forward Looking Statements

This press release contains forward-looking statements based on Sangamo’s current expectations. These forward-looking statements include, without limitation, statements relating to Sangamo’s zinc finger proteins’ potential to be used with Chroma’s epigenetic editors, the potential for Chroma to develop epigenetic medicines leveraging ZFPs for sequence-specific DNA recognition, the potential for Sangamo to provide Chroma specific ZFPs designed for collaboration targets outside of the central nervous system and for Sangamo to receive an upfront technology access payment, the potential for Sangamo to design and engineer ZFPs to provide valuable capability beyond what is available with competing technologies, the potential for Chroma to exercise its option and for Sangamo to receive development and commercial milestone payments and royalties. These statements are not guarantees of future performance and are subject to certain risks and uncertainties that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, the research development process, including the results of clinical trials; the regulatory approval process for product candidates; and the potential for technological developments that obviate technologies used by Sangamo and its partners, the COVID-19 pandemic; the potential for Chroma to breach or terminate its agreement with Sangamo; and the potential for Sangamo to fail to realize its expected benefits from the Chroma agreement, including but not limited to further validating the importance of the zinc finger platform to support epigenetic editing. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamo’s operations and business. These risks and uncertainties are described more fully in our Securities and Exchange Commission filings and reports, including in our Annual Report on Form 10-K for the year ended December 31, 2022 and our Quarterly Report on Form 10-Q for the quarter ended March 31, 2023. Forward-looking statements contained in this announcement are made as of this date, and Sangamo undertakes no duty to update such information except as required under applicable law.

Sangamo Contact

Investor Relations & Media Inquiries
Louise Wilkie
ir@sangamo.com
media@sangamo.com 


Chroma Contact

Lia Dangelico
Verge Scientific Communications
540.303.0180
ldangelico@vergescientific.com

Boston, MA, May 19, 2023Chroma 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) 26th 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 19th, 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

Boston, MA, May 18, 2023Chroma Medicine, Inc., (Chroma) a genomic medicine company pioneering single-dose epigenetic editing therapeutics, today announced in vivo data that demonstrate 99% silencing of two distinct targets and compelling proof-of-concept for its epigenetic editing platform. The data presented at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting showcase the rapid advancement of the company’s programs and platform.

The work describes the development of Chroma’s epigenetic editor targeting PCSK9, a well-characterized genetic target for addressing hyperlipidemia. The company’s epigenetic editor showed highly effective, durable, and specific silencing of PCSK9 in a transgenic mouse. Ninety-nine percent silencing of PCSK9 was observed following a single dose of Chroma’s epigenetic editor through a minimum of five months of observation with no detectable off-target changes in gene expression or methylation.

In addition, Chroma’s presentation highlighted the broad utility and modularity of its epigenetic editors. Data shared demonstrate that exchanging the PCSK9 targeting guide RNA with one directed towards the HBV genome induced robust and durable reduction of hepatitis B surface antigen (HBsAg) below the lower limit of quantification following a single administration in a transgenic HBV mouse model.

“These data illustrate the inherent power of epigenetic editing. The robust efficiency, durability, and specificity of silencing observed at two distinct targets underscore the advantages of harnessing an endogenous mechanism for gene regulation,” said Vic Myer, Ph.D., Chroma’s President and CSO. “In combination with our multiplex editing data—and data from the lab of our scientific co-founder Dr. Angelo Lombardo also presented at ASGCT—the results underscore the transformative potential of epigenetic editing as the modality of choice for gene regulation.”

The company expects to present additional data at upcoming scientific meetings, signaling advancement of its broad portfolio of highly differentiated epigenetic editing therapeutics for patients living with serious diseases.

Details for Chroma’s oral presentations at ASGCT 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 18th, 2:30p PT
Session:  Gene Targeting and Gene Correction: Liver

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


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

# # #

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, programmable, 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

Boston, MA, May 2, 2023Chroma 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) 26th 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 18th, 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 19th, 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.

Media Contact

Lia Dangelico
Verge Scientific Communications
540.303.0180
ldangelico@vergescientific.com