CELL / GENE THERAPIES & RARE AND METABOLIC DISEASES
- 30 years of hands-on experience as a specialist in developing small molecules, biologics, and cellular therapies for rare genetic diseases, and metabolic and orphan diseases.
- Excels in seed early-stage, venture-backed opportunities.
- Broad expertise in leadership; basic, preclinical, and clinical research; clinical development; medical affairs; post-marketing development; and regulatory strategy across all agencies.
- Skilled in defining, communicating, and executing research, clinical, regulatory, and commercial strategies.
CMO, Chief Development Officer, Senior Clinical Advisor, DSMB Member, Research, Clinical Development, Medical Affairs, Regulatory Strategy, and Post-marketing Development. Therapeutic Areas: Rare Diseases, Hematology, Neurology, Immunology, Inflammation, Dermatology, Medical/Psychiatric Disorders, and Palliative Care (anxiety).
Managing Director, Mirror Neuron Partners, LLC (2005 – present)
Medical consultant to biopharma, private equity funds, governmental and other organizations.
Consultant, AVROBIO (2021 – present)
Counsels team on development of gene therapies for Fabry program.
Senior Advisor, Eloxx Pharmaceuticals (formerly Zikani) (2019 – present)
Provides strategic advice to Senior Management for development of therapeutics using proprietary aminoglycoside and macrolide platforms in the treatment of genetic diseases caused by non-sense mutations.
Chief Medical Officer (CMO), Inozyme Pharma, Inc. (2019 – 2021)
Led translational, medical, clinical, and regulatory efforts for development of therapeutics for disorders of pathological mineralization for treatment of genetic diseases caused by non-sense mutations. Responsible for world-wide development efforts across multiple therapeutic modalities and indications.
Chief Medical Officer (CMO), Sentien Biotechnologies, Inc. (2018 – 2019)
Led translational, medical, clinical, and regulatory efforts for development of applications of mesenchymal stromal cells in diseases of dysregulated inflammation.
Chief Medical Officer (CMO), Eloxx Pharmaceuticals (2015 – 2018)
Led translational, medical, clinical, and regulatory efforts to develop advanced designer aminoglycosides for treatment of genetic diseases caused by non-sense mutations. Responsible for world-wide development efforts across multiple therapeutic areas and indications. Part of team that took company public (NASDAQ: ELOX).
Precision Medicine Clinical Lead (2014-2015) and Global Lead (2012), Pfizer
Oversaw translational medicine efforts for rare diseases, as well as programs in hematology, neurology, immunology, inflammation, and dermatology. Served as Medicines Development Group Lead (2012-2014). Headed worldwide development and medical affairs in the TTR-amyloidosis for the polyneuropathy indication in TafamidisÒ program. Led opportunities in rare neurodegenerative disorders. Spearheaded global medical affairs in the TTR-amyloidosis for the polyneuropathy indication in TafamidisÒ program.
Global Medical Lead, Shire Human Genetic Therapies (2008 – 2011)
Headed medical/clinical areas of rare diseases franchise and led registration effort of Fyrazirä.
Chief Development Officer, Advanced Cell Technology (2007 – 2008)
Designed/directed corporate strategies for therapeutic embryonic and adult stem cells. Formed regulatory, clinical, and manufacturing departments; initiated /directed regulatory interactions with FDA; and formulated/implemented positioning and development strategy for products in three indication areas. Solved scientific basis for human embryonic stem cells developing into human pigmented retinal epithelium.
Chief Strategy and Development Officer, Amicus Therapeutics (2005 – 2006)
Designed, implemented, and managed product development pathway for pharmacological chaperones, including corporate vision and planning; budget and tactics; and R&D, preclinical/clinical development activities, and KOL network.