Finding cures for children's genetic diseases


CMRI expertise sought internationally

We’re thrilled to announce that Dimension Therapeutics, Inc. a leading rare disease company advancing novel, liver-directed treatments for diverse genetic disorders, has recently expanded its Scientific Advisory Committee with the appointment of Children’s Medical Research Institute’s Head of the Gene Therapy Research Unit, Ian Alexander, M.B.B.S., Ph.D. 

Dimension’s Chief Executive Officer Annalisa Jenkins, MBBS, MRCP says;
 “Dimension has attained significant momentum, and we are able to access a leading group of advisors as we prepare to commence our first clinical trial in the second half of this year and advance additional programs to meet compelling unaddressed needs for patients with rare diseases

 “We are thrilled to attract the leading researchers and clinical experts to our science advisory group, as well as to our newly formed advisory boards for our programs in OTC deficiency, the most common urea cycle disorder, and GSDIa.

Dimension Therapeutics, Inc are advancing multiple programs toward clinical development, including: programs addressing unmet needs for patients suffering from OTC deficiency and GSDIa; a collaboration with Bayer HealthCare in hemophilia A, and a wholly owned program in hemophilia B, which is expected to enter clinical testing in the second half of 2015. Dimension has preferred access to multiple best-in-class AAV vectors from REGENXBIO, which founded Dimension with Fidelity Biosciences in October 2013. The Dimension team and senior advisors include biotech industry veterans and renowned thought leaders in gene therapy and rare diseases. 

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