Audentes Therapeutics Announces Significant Additions to Leadership Team

SAN FRANCISCO--(BUSINESS WIRE)--Audentes Therapeutics, Inc., a biotechnology company dedicated to the development and commercialization of innovative treatments for rare diseases using gene therapy technology, has announced the expansion of its leadership team, including the appointment of Suyash Prasad, M.D. as Senior Vice President and Chief Medical Officer. Dr. Prasad is the latest addition to a management group with significant experience and a history of success in the industry. In addition to Dr. Prasad, Audentes recently appointed Michael O'Callaghan, D.V.M., Ph.D. as Senior Vice President, Preclinical Development and Translational Medicine, Barbara Wuebbels, RN as Vice President, Patient Advocacy and Medical Affairs, and Monica Miller as Vice President, Program and Alliance Management.

"In just a matter of months we have built a world-class team of talented and passionate individuals committed to ensuring the availability of new treatments for patients with serious, rare diseases," commented Matthew Patterson, President and Chief Executive Officer of Audentes Therapeutics. "The addition of Dr. Prasad and the rest of the team ensures we will rapidly advance our lead programs in X-Linked Myotubular Myopathy and Pompe disease and creates an outstanding foundation for the future growth of Audentes."

Dr. Prasad joins Audentes after 12 years in positions of increasing responsibility at BioMarin Pharmaceutical, Inc., Genzyme Corporation and Eli Lilly and Company. He has a wide range of experience and achievement in international drug development across Phase I to IV, with a specific focus in the development of therapies to treat rare pediatric disorders. Dr. Prasad's areas of expertise include neuromuscular disease, metabolic medicine, bone, neuroscience, endocrinology, immunology, and genetics. He has significant experience with the development and commercialization of enzyme replacement therapies to treat lysosomal storage disorders, including Cerezyme®, Aldurazyme®, Fabrazyme®, and Myozyme®. Dr. Prasad was most recently responsible for the clinical development of novel treatments for phenylketonuria (PKU) and achondroplasia. Dr. Prasad graduated in Medicine at the University of Newcastle-upon-Tyne, UK, and is a United Kingdom board certified physician with a sub-specialty interest in Pediatric Critical Care. He is a member of the Royal College of Physicians (MRCP), the Royal College of Pediatrics and Child Health (MRCPCH), and the Faculty of Pharmaceutical Medicine (MFPM).

Dr. O'Callaghan joined Audentes following 15 years of holding executive leadership positions related to research and development at Genzyme Corporation. He was responsible for therapeutic development strategies and assisting with regulatory strategy for several rare disease therapeutics, most notably Myozyme®, Fabrazyme®, and Carticel®. Apart from the orphan disease portfolio, he also worked on cardiovascular, orthopedic and oncology indications employing several different technology platforms, including proteins, biopolymers, cell therapies, and small molecule drugs. Prior to Genzyme, Dr. O'Callaghan was an academic and clinical veterinary radiologist, first at Massey University in New Zealand for 8 years, then at Tufts University School of Veterinary Medicine for 10 years, the last 3 as Head of Department.

Ms. Wuebbels, RN joined Audentes following 6 years at BioMarin Pharmaceutical, Inc. in leadership positions related to patient advocacy and medical affairs. This work included the creation of a global patient advocacy and investigator relations department. Through this new department, Ms. Wuebbels linked the critical perspectives of patient advocates with the internal planning of clinical and commercial activities. She also had oversight of BioMarin's global registry for Maroteaux-Lamy Syndrome (MPS VI) patients. Prior to BioMarin, Ms. Wuebbels served for 8 years as Director of Clinical Education for the orphan drug division of Medicis Corporation and prior to that served in multiple positions related to clinical research. She started her career as a registered nurse.

Ms. Miller spent over 9 years at BioMarin Pharmaceutical, Inc. prior to joining Audentes. While there, Ms. Miller served in positions of increasing responsibility in product development and project management. Most recently, Ms. Miller served as program leader for the development of Vimizim®, the recently-approved treatment for patients with MPS IVA or Morquio Syndrome. In this role, she oversaw the program from preclinical through clinical development to license application, including commercial planning activities. Ms. Miller was also part of the alliance management team for external patient organizations. Prior to BioMarin, Ms. Miller worked in project management for a major, federally-funded genomics research project, as well as a research laboratory manager at a genomics company.

About X-Linked Myotubular Myopathy (XLMTM)

X-Linked Myotubular Myopathy (XLMTM) is a rare, inherited disorder characterized by severe muscle weakness and respiratory impairment. It is caused by mutations in the MTM1 gene, which encodes an enzyme called myotubularin. Myotubularin is thought to be involved in the development and maintenance of muscle cells. XLMTM affects 1 in 50,000 newborn males worldwide. In a majority of newborns with the disorder, XLMTM causes death in the first years of life. There is currently no effective treatment for this disease. Audentes is developing AT001 for the treatment of XLMTM in collaboration with Genethon ( AT001 is a novel product based on AAV gene therapy technology.

About Pompe Disease

Pompe Disease is a rare, inherited disorder characterized by progressive muscle weakness and respiratory impairment. It is caused by mutations in a gene that encodes an enzyme called acid alpha-glucosidase (GAA), which is needed by the body to break down glycogen – a stored form of sugar used for energy. Pompe Disease affects approximately 1 in every 40,000 births. Audentes is developing AT002 for the treatment of Pompe disease. AT002 is a novel product based on AAV gene therapy technology.

About Audentes Therapeutics, Inc.

Audentes™ is a private biotechnology company committed to the development and commercialization of innovative new treatments for people with serious, rare diseases through the application of gene therapy technology. The company consists of a focused, experienced, and passionate team driven by the goal of improving lives of patients. Audentes takes pride in strong, global relationships with the patient, research, and medical communities.

For more information regarding Audentes, please visit


Audentes Therapeutics, Inc.
Jeffrey Gruis, 415-638-6561
[email protected]