Dimension Therapeutics Announces Presentation at the 58th American Society of Hematology (ASH) Annual Meeting

Researchers report the importance of capsid selection in AAV vector design and demonstrate that Clade E vectors have the highest levels of periportal liver gene transfer versus other serotypes


CAMBRIDGE, Mass., Dec. 05, 2016 (GLOBE NEWSWIRE) -- Dimension Therapeutics, Inc. (NASDAQ:DMTX), a biopharmaceutical company advancing novel, adeno-associated virus (AAV) gene therapies targeting the liver, a key organ for human metabolism, today announced an oral poster presentation that highlights findings of in vivo preclinical studies demonstrating that the selection of capsid, a key component of AAV vectors, can enhance product efficiency and potentially reduce host immune responses to therapy. The presentation took place during the 58th American Society of Hematology (ASH) Annual Meeting, which is being held in San Diego, California, December 3-6, 2016.

In the poster, titled “Strategies for Selection of AAV Vectors for Administration to Liver: Studies in Nonhuman Primates” (Abstract # 2316), researchers at the University of Pennsylvania (Penn) and Dimension are reporting that capsid serotypes AAV8 and AAVrh10, both members of the Clade E AAV family, result in higher levels of gene transfer to the primate liver than other vectors, including AAV5 and AAV3B. In addition, the choice of capsid can result in reduced production of neutralizing antibodies as part of the host immune response and potentially limit immune responses to follow-on administration of a second vector.

“We are pleased to announce key findings from our highly productive collaboration with Penn and the lab of James Wilson, MD, PhD, a professor of Medicine and Pediatrics in the Perelman School of Medicine at Penn,” said Dr. Annalisa Jenkins, MBBS, FRCP, Chief Executive Officer of Dimension. “These data add to the body of science supporting Dimension’s choice of the Clade E family of capsid serotypes for programs where the liver is the primary target organ.”

Penn, Wilson, and Wadsworth are each holders of Dimension stock, and Wilson also holds stock in REGENXBIO, Inc. Dimension holds exclusive sublicenses from REGENXBIO, Inc. for certain Penn-owned AAV technologies related to this development program and has an ongoing research collaboration and licensing relationship with Penn.

About Dimension Therapeutics, Inc.

Dimension Therapeutics, Inc. (NASDAQ:DMTX) is the leader in discovering and developing new therapeutic products for people living with devastating rare and metabolic diseases associated with the liver, based on the most advanced, mammalian adeno-associated virus (AAV) gene delivery technology. Dimension is actively progressing its broad pipeline, which features programs addressing unmet needs for patients suffering from inherited metabolic diseases, including OTC deficiency, GSDIa, citrullinemia type 1, PKU, Wilson disease, a collaboration with Bayer in hemophilia A, and a wholly owned clinical program in hemophilia B. The company targets diseases with readily identifiable patient populations, highly predictive preclinical models, and well-described, and often clinically validated, biomarkers. Founded in 2013, Dimension maintains headquarters in Cambridge, Massachusetts.

For more information, please visit www.dimensiontx.com.

Cautionary Note Regarding Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding development, preclinical results, and the continued progress of Dimension’s portfolio and program. All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include the risks that Dimension’s product candidates, including its lead candidate, DTX101, will not successfully be developed or commercialized; and the risks described under the caption "Risk Factors" in Dimension Therapeutics’ Quarterly Report on Form 10-Q for the quarter ended September 30, 2016, which is on file with the Securities and Exchange Commission, as well as other risks detailed in Dimension Therapeutics’ additional filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Dimension Therapeutics undertakes no duty to update this information unless required by law.


            

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