In Vivo Preclinical Studies Support a Promising Profile for Lead Candidate Currently in IND-Enabling Studies
CAMBRIDGE, MA, USA I May 07, 2016 I Dimension Therapeutics, Inc. (DMTX), a biopharmaceutical company advancing novel, adeno-associated virus (AAV) gene therapies targeting the liver, a central organ for devastating rare diseases, today announced positive preclinical results from its partnered development program with Bayer. Preclinical data from the partnered hemophilia A program were highlighted in a poster, titled “Optimized AAV-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice and Cynomolgus Macaques” (Abstract No. 760), presented at the 19th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, which is taking place in Washington, DC, May 4-7, 2016.
Initiated in June 2014, Bayer and Dimension’s collaboration is advancing in IND-enabling studies a novel AAV gene therapy product, DTX201, to treat patients with hemophilia A. Under the agreement, Dimension is responsible for all development activities through Phase 1/2a clinical testing, with funding provided by Bayer. Depending on the results of the Phase 1/2a clinical trial, Bayer will conduct the confirmatory Phase III clinical study, submit all regulatory filings, and be responsible for commercialization. Bayer has worldwide rights to commercialize any potential future product for the treatment of hemophilia A developed with Dimension, which is eligible to receive tiered royalties based on product sales.
Preclinical research conducted with Dimension’s collaborators at the University of Pennsylvania (“PENN”) demonstrated that the selection and combination of specific product components – including the capsid, enhancer, and promoter – further optimized product performance, including long-term expression of Factor VIII (FVIII), the protein missing or deficient in hemophilia A that DTX201 is designed to provide. The FVIII expression levels, achieved during the 30-week course of the study, supported advancement of DTX201 into IND-enabling studies for the potential treatment of patients with hemophilia A.
“As a leader in hemophilia and evolving new treatment approaches for patients, Bayer has been an ideal partner for us to pursue our program in hemophilia A,” said Dr. Annalisa Jenkins, MBBS, FRCP, Chief Executive Officer of Dimension. “We continue to benefit greatly from our research collaboration with PENN that has enabled the delivery of high quality in vivo studies to support our Hemophilia A program.”
PENN and Dr. James M. Wilson 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.
About DTX201
Dimension is developing its gene therapy product DTX201 in partnership with Bayer for the treatment of hemophilia A. DTX201 is designed to deliver Factor VIII, or FVIII, gene expression in a durable fashion, potentially obviating the need for long-term routine intravenous injections of hemophilia A patients. Preclinical studies completed to date indicate DTX201 has the potential to be a well-tolerated, effective therapy for hemophilia A. DTX201 is currently in IND-enabling studies.
About Dimension Therapeutics, Inc.
Dimension Therapeutics, Inc. (DMTX) is the leader in discovering and developing new therapeutic products for people living with devastating rare 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 and GSDIa, 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.
SOURCE: Dimension Therapeutics
Post Views: 278
In Vivo Preclinical Studies Support a Promising Profile for Lead Candidate Currently in IND-Enabling Studies
CAMBRIDGE, MA, USA I May 07, 2016 I Dimension Therapeutics, Inc. (DMTX), a biopharmaceutical company advancing novel, adeno-associated virus (AAV) gene therapies targeting the liver, a central organ for devastating rare diseases, today announced positive preclinical results from its partnered development program with Bayer. Preclinical data from the partnered hemophilia A program were highlighted in a poster, titled “Optimized AAV-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice and Cynomolgus Macaques” (Abstract No. 760), presented at the 19th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, which is taking place in Washington, DC, May 4-7, 2016.
Initiated in June 2014, Bayer and Dimension’s collaboration is advancing in IND-enabling studies a novel AAV gene therapy product, DTX201, to treat patients with hemophilia A. Under the agreement, Dimension is responsible for all development activities through Phase 1/2a clinical testing, with funding provided by Bayer. Depending on the results of the Phase 1/2a clinical trial, Bayer will conduct the confirmatory Phase III clinical study, submit all regulatory filings, and be responsible for commercialization. Bayer has worldwide rights to commercialize any potential future product for the treatment of hemophilia A developed with Dimension, which is eligible to receive tiered royalties based on product sales.
Preclinical research conducted with Dimension’s collaborators at the University of Pennsylvania (“PENN”) demonstrated that the selection and combination of specific product components – including the capsid, enhancer, and promoter – further optimized product performance, including long-term expression of Factor VIII (FVIII), the protein missing or deficient in hemophilia A that DTX201 is designed to provide. The FVIII expression levels, achieved during the 30-week course of the study, supported advancement of DTX201 into IND-enabling studies for the potential treatment of patients with hemophilia A.
“As a leader in hemophilia and evolving new treatment approaches for patients, Bayer has been an ideal partner for us to pursue our program in hemophilia A,” said Dr. Annalisa Jenkins, MBBS, FRCP, Chief Executive Officer of Dimension. “We continue to benefit greatly from our research collaboration with PENN that has enabled the delivery of high quality in vivo studies to support our Hemophilia A program.”
PENN and Dr. James M. Wilson 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.
About DTX201
Dimension is developing its gene therapy product DTX201 in partnership with Bayer for the treatment of hemophilia A. DTX201 is designed to deliver Factor VIII, or FVIII, gene expression in a durable fashion, potentially obviating the need for long-term routine intravenous injections of hemophilia A patients. Preclinical studies completed to date indicate DTX201 has the potential to be a well-tolerated, effective therapy for hemophilia A. DTX201 is currently in IND-enabling studies.
About Dimension Therapeutics, Inc.
Dimension Therapeutics, Inc. (DMTX) is the leader in discovering and developing new therapeutic products for people living with devastating rare 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 and GSDIa, 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.
SOURCE: Dimension Therapeutics
Post Views: 278
