Fidelity Biosciences and REGENX Biosciences Launch Dimension Therapeutics to Develop and Commercialize Novel AAV Gene Therapy Products

CAMBRIDGE, MASS. & WASHINGTON — Fidelity Biosciences and REGENX Biosciences today announced the formation of Dimension Therapeutics, a gene therapy company focused on developing novel treatments for rare diseases. Dimension will focus on advancing its platform of gene therapy programs in rare diseases through clinical development, starting with lead programs in hemophilia, and building out a world-class product engine for AAV therapeutics. Dimension has completed an undisclosed Series A financing led by Fidelity Biosciences.

In conjunction with its launch, Dimension has entered into an exclusive license and collaboration with REGENX. REGENX holds exclusive rights to a portfolio of over 100 patents and patent applications pertaining to its NAV® vector technology that includes novel AAV vectors such as rAAV7, rAAV8, rAAV9, and rAAVrh10. Through its license and collaboration with REGENX, Dimension has acquired preferred access to NAV vector technology and rights in REGENX product programs in multiple rare disease indications.

"Gene therapy is a fundamental method of disease intervention, changing a patient's genetic code to treat genetic disease, and in some cases providing a potential lifelong benefit following a single treatment," said Thomas R. Beck, M.D., executive partner at Fidelity Biosciences and interim chief executive officer of Dimension Therapeutics. "A core challenge for gene therapy has been the development of safe, efficient 'vectors' to enable delivery of the replacement gene to the correct cells and tissues of the patient to yield benefit. We believe REGENX NAV vectors are the most promising approach for in vivo gene therapy and represent the potential for transformative therapy for patients."

Dimension has assembled a team of leaders in the areas of rare disease and gene therapy as well as industry veterans and experienced entrepreneurs. The company has appointed Dr. Beck as interim chief executive officer and Sam Wadsworth, Ph.D., as chief scientific officer. Dr. Wadsworth was previously head of gene therapy research and development at Genzyme, where he led preclinical development for multiple rare disease and gene therapy programs.

The company's scientific advisors are leading experts in the field of gene therapy and rare disease. Dr. James Wilson, director of the gene therapy program at the University of Pennsylvania and the scientific founder of REGENX, will chair the company's Scientific and Technical Advisory Board. NAV vector technology was discovered in the laboratory of Dr. Wilson at the University of Pennsylvania. Other advisors to Dimension include Emil D. Kakkis, M.D., Ph.D., president and chief executive officer of Ultragenyx, a leading rare disease company, and former chief medical officer of Biomarin.

Ben Auspitz, partner at Fidelity Biosciences, has been appointed chairman of the Board of Dimension, and will be joined by directors Allan M. Fox, founding and managing partner of FOXKISER, the entrepreneurial force behind REGENX; Donald J. Hayden, an experienced pharmaceutical executive and the chairman of REGENX; and Dr. Beck.

In parallel to the formation of Dimension, Fidelity Biosciences has also made a direct investment into REGENX, with Mr. Auspitz joining the REGENX Board.

"We are pleased to work with Fidelity to establish a new best-in-class company in AAV gene therapy that has the opportunity to invest in the focused development of multiple important rare diseases, including hemophilia," said Ken Mills, president and chief executive officer of REGENX. "We view the formation of Dimension as important in the evolution of REGENX's mission to enable access to NAV vector technology through partnership and licensing to create successful new AAV therapeutics."

About Fidelity Biosciences

Fidelity Biosciences ( invests venture capital in biopharmaceutical, medical technology, healthcare information technology and healthcare service companies. The firm is a subsidiary of FMR LLC, the parent company of Fidelity Investments, one of the world's leading providers of financial services. For more than 40 years, Fidelity has been a significant presence in the venture capital and private equity industry. For more information, visit the website above.

About REGENX Biosciences

REGENX Biosciences is the leading AAV gene therapy company that is developing a new class of personalized therapies, based on its proprietary NAV vector technology platform, for a range of severe diseases with serious unmet needs. NAV vector technology includes novel AAV vectors such as rAAV7, rAAV8, rAAV9, and rAAVrh10. Our treatments in development include programs for hypercholesterolemia, mucopolysaccharidoses, and retinitis pigmentosa. REGENX leadership in AAV gene therapy and corresponding intellectual property has enabled it to establish collaborations with leading global partners including Chatham Therapeutics, Fondazione Telethon, and Audentes Therapeutics. In addition, together with Fidelity Biosciences, REGENX has formed Dimension Therapeutics a company focused on the development and commercialization of AAV gene therapies for rare diseases. For more information regarding REGENX, please