Fondation Ipsen

Rare Disease : Innovations in Diagnostics and Therapeutics (part 2)

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Discover the second cycle of 4 conferences from the “Innovations in Diagnostics and Therapeutics: An Interdisciplinary Virtual Rare Disease Symposium”. This conference, online and free, was organized by UCSF – Innovation Ventures and the Fondation Ipsen.


Teaser of the second cyle:



Conference 1

Negotiating the Challenges of Therapeutics Discovery for Rare Diseases

By Emil D. Kakkis, MD, PhD (Founder, President, and CEO, Ultragenyx)

Emil D Kakkis, MDcis a medical geneticist by training, Dr. Kakkis is a pioneer in the development and commercialization of treatments for rare diseases. He is founder and CEO at Ultragenyx, a biopharmaceutical company developing novel products for the treatment of rare and ultra-rare diseases. Before founding Ultragenyx in 2010, Dr. Kakkis guided and contributed to the development of multiple approved rare disease products
as chief medical officer at BioMarin Pharma- ceutical. Dr. Kakkis also founded and serves on the Board of EveryLife Foundation for Rare Diseases, a non-profit dedicated to accelerating innovation for rare diseases. Dr. Kakkis has been recognized for his leadership by Biotechnology Innovation Organization (BIO), the National MPS Society, WORLDSymposium, and California Life Sciences.


Conference 2

n-Lorem: Meeting the Needs of n-1 Patients One Patient at a Time

By Stanley Crooke, MD, PhD (Founder and CEO, n-Lorem)

Dr. Stanley Crooke is a founder, chairman and chief executive officer of n-Lorem, a nonprofit foundation focused on providing treatments for patients with ultra-rare diseases. Prior to nLorem, Dr. Crooke founded Ionis Pharmaceuticals. During his tenure at Ionis, he has led the scientific development of a new platform for drug discovery, antisense technology and engineered the creation of one of the largest and more advanced development pipelines in the biotechnology industry. Early in Dr. Crooke’s career, he led the creation of the first broad anticancer program in the industry at Bristol- Myers, bringing numerous anticancer drugs to the market. He went on to be responsible for worldwide R&D at SmithKline Beckman.

Dr. Crooke has received several awards including the American Chemical Society’s E.B. Hershberg Award for Important Discoveries in Medicinally Active Substances, the Lifetime Achievement Award presented by the Oligonucleotide Thera- peutics Society, and the Scrip Lifetime Achieve- ment Award. Dr. Crooke received his MD and PhD degrees at Baylor College of Medicine.

In 2021, Dr. Crooke was named Distinguished Alumnus of both Baylor College of Medicine’s Graduate and Medical schools.


Conference 3

Defining a New Future for People with Rare Diseases

By Amrit Ray, MD, MBA (Chief Patient Officer, Biohaven Pharmaceuticals)

A globally-experienced industry leader, physician researcher, and biopharmaceuti- cals expert, Dr. Amrit Ray’s passion is advancing medical breakthroughs and championing healthcare access for patients. He serves as a board director and independent advisor to biopharmaceutical companies. Dr.Ray has held three C-level roles at major companies including as Global President of R&D at Pfizer and as Chief
Medical Officer at Johnson & Johnson pharmaceuticals. Most recently, Dr.Ray was an Executive Leadership Team member at Pfizer-Upjohn overseeing all aspects of R&D worldwide and responsibility as the company’s most senior decision-maker on patient matters. He has led teams of up to 3,500 professionals in 90 countries to advance pipelines and access for innovative medicines in Oncology, Neuro- science and Cardio-Metabolism, and sponsored >100 pioneering inter-sectoral healthcare partnerships.

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