Pursuing Hope: How The Commercialization of Spinraza Has Changed Lives

Wednesday, February 21 

Moderator: Laura Savatski - Executive Director of Innovation, University of Louisville

Panelists:

Andrew Whiteley – VP Business Development and Technology Transfer at Cold Spring Harbor Laboratory
Adrian Krainer, PhD – St. Giles Foundation Professor, Cancer Center Deputy Director of Research, Cold Spring Harbor Laboratory
C. Frank Bennett, PhD – Executive Vice President, Chief Scientific Officer, Ionis Pharmaceuticals, Inc.

Follow the journey that led to the development and commercialization of a life-saving treatment for Spinal Muscular Atrophy, one of the deadliest inherited diseases in children under the age of two. Starting with a scientist’s relentless pursuit for a cure, fueled by a nonprofit’s commitment to empowering patients, learn how the Cold Spring Harbor Laboratory’s tech transfer office partnered with pharmaceutical giant Biogen and others to solve the unsolvable and bring hope to families around the globe.

Moderator

Laura Savatski has a diverse background as a research scientist, entrepreneur, and start-up advisor, and broad experience bringing inventions to market. Laura's early career in medical research focused on vaccine trials, molecular virology, stem cell biology, transplant/oncology, and cellular assays. Her past roles include Vice President and Chief Operating Officer for Prodesse, a company she co-founded, which make molecular infectious disease diagnostic products and is now part of Hologic. Laura has served as the AUTM Board representative for the Alliance of Technology Transfer Professionals (ATTP) the organization that awards the Registered Technology Transfer Professional (RTTP) credential for the profession and recently served as the AUTM Chair.

About the Speakers

Andrew Whiteley is VP Business Development and Technology Transfer at Cold Spring Harbor Laboratory. Whiteley’s broad experience in commercializing life science technologies supports the commercial spin-outs from Cold Spring Harbor Laboratory. He serves new companies in roles ranging from director to mentor to consultant on issues related to business strategy, fundraising and organization development.

With a chemistry and biochemistry degree from the University of Nottingham, Whiteley started his career at Amersham International (now part of GE Healthcare) as a biochemist and went on to hold sales, marketing and strategic business management positions, becoming VP Sequencing and leading a cross-functional team commercializing DNA Sequencing Systems. He was the CEO of two biotech companies: InforMax Inc., where he reorganized the company before selling it to Invitrogen (now part of ThermoFisher Scientific); and Vitra Biosciences.


 C. Frank Bennett, PhD, is the executive vice president and chief scientific officer at Ionis Pharmaceuticals and one of the founding members of the company. He is responsible for continuing to advance Ionis’ technology and expanding the company’s drug discovery platform. Bennett is also the franchise leader for gene-editing programs at Ionis. He has been involved in the development of antisense oligonucleotides as therapeutic agents, including research on the application of oligonucleotides for inflammatory, neurodegenerative diseases and cancer, oligonucleotide delivery, pharmacokinetics and medicinal chemistry.
 
Bennett has been recognized for his research with numerous awards. He is a co-recipient of the Rainwater Prize for Outstanding Innovation in Neurodegenerative Disease Research, the 2021 Gabbay Award and the 2019 Breakthrough Prize in Life Sciences for his contributions to the discovery and development of SPINRAZA® (nusinersen), and the inaugural Healy Center International Prize for Innovation in amyotrophic lateral sclerosis (ALS). Bennett also received the 2018 Hereditary Disease Foundation's Leslie Gehry Brenner Prize for Innovation in Science for his leadership and continued commitment to developing antisense therapies for Huntington's disease.


Adrian Krainer, PhD, is a biochemist recognized for his basic work on RNA splicing and the development of mechanism-based therapeutic applications. Krainer’s lab studies the mechanisms of RNA splicing, ways in which they go awry in cancer and genetic diseases, and the means by which faulty splicing can be corrected. For example, they study splicing in spinal muscular atrophy (SMA), a neuromuscular disease that is the leading genetic cause of death in infants.

In SMA, a gene called SMN2 is spliced incorrectly, making it only partially functional. The Krainer lab found a way to correct this defect using a powerful therapeutic approach. It is possible to stimulate SMN protein production by altering mRNA splicing through the introduction into cells of chemically modified pieces of RNA called antisense oligonucleotides (ASOs). Following extensive work with ASOs in mouse models of SMA, one such molecule, known as nusinersen or Spinraza, was taken to the clinic, and at the end of 2016 it became the first FDA-approved drug to treat SMA, by injection into the fluid surrounding the spinal cord.

Krainer is a recipient of the Life Sciences Breakthrough Prize, the RNA Society’s Lifetime Achievement Award, the Reemtsma Foundation International Prize in Translational Neuroscience, the Speiser Award in Pharmaceutical Sciences and the Ross Prize in Molecular Medicine. He served as President of the RNA Society and is a member of the National Academy of Sciences, the National Academy of Medicine, the National Academy of Inventors and the American Academy of Arts & Sciences.