Rainwater Charitable Foundation Announces Fourth-Annual Rainwater Prize Winners for Brain Research

Honorees are recognized for their innovative scientific discoveries for neurodegenerative diseases

FORT WORTH,Texas,Jan. 26,2023 -- The Rainwater Charitable Foundation,one of the largest independent funders of neurodegenerative disease research,today announced this year's recipients of the Rainwater Annual Prize for Outstanding Innovation in Neurodegenerative Disease Research and for Innovative Early Career Scientist. This year's Outstanding Innovation Prize will be awarded to a team: Dr. C.Frank Bennett,Ionis; Dr. Don W. Cleveland,University of California San Diego (UCSD); and Dr. Timothy M. Miller,Washington University. Dr. Susanne Wegmann,German Center for Neurodegenerative Diseases (DZNE) will be awarded the Rainwater Prize for Innovative Early-Career Scientist. The prizes will be presented during the Eurotau 2023 Conference on April 27,2023 in Lille,France.

The Rainwater Prize Programrecognizes scientific progress toward new treatments for neurodegenerative diseases associated with the accumulation of tau protein in the brain and fosters scientific discovery by elevating awareness of the gaps in neurodegenerative research,bringing new researchers into the tauopathy field,and awarding scientific achievements that could lead to innovative,effective treatments.

Rainwater Prize for Outstanding Innovation in Neurodegenerative Disease Research will be shared by:

Collaborators Drs. Bennett,Cleveland and Miller for their work advancing antisense technology for the treatment of neurodegenerative diseases including ALS,Huntington's disease,Alzheimer's disease and tauopathies over the past 20 years.

Dr. Bennett,Chief Scientific Officer at Ionis,continues to advance antisense technology and expand Ionis's drug discovery platform. He has been working on antisense oligonucleotides for the past 30 years. He started investigating the potential of antisense technology for developing therapies for ALS and spinal muscular atrophy (SMA) almost 20 years ago. The first drug for the treatment of SMA,nusinersen,was approved by the FDA in 2016 and subsequently has been approved in more than 50 countries world-wide. He is currently working on a treatment for Huntington's disease,which is in late-stage clinical trials and is leading the discovery and development of other drugs for neurological diseases.

Dr. Cleveland,Chair and Distinguished Professor of Cellular and Molecular Medicine at UC San Diego,discovered,purified,and determined the properties of the tau protein,the major component of the intracellular filamentous deposits that define a number of neurodegenerative diseases including Progressive Supranuclear Palsy. With Bennett and Miller,he developed designer DNA drugs that utilize technology that silence genes in the human nervous system that have been studied in seven different clinical trials,aiming to treat ALS,Huntington's Disease,Parkinson's Disease,and Alzheimer's Disease.

Dr. Miller,Vice Chair of Research and David Clayson Professor of Neurology at Washington University School of Medicine in St. Louis,has been a leader for over two decades in helping uncover effective therapies for neurodegenerative diseases including ALS and tauopathies. In 2007,Dr. Miller created an ALS translational research program and launched promising trials to improve the lives of people with neurological disorders. The latest results using the SOD1 ASO developed with Cleveland and Bennett show a dramatic effect on slowing of SOD1 ALS. His lab led the early stages of development of BIIB080/IONIS-MAPTRx,an antisense drug that lowers tau mRNA and protein as a therapy for tauopathies.

"This prize is an honor and I look forward to using this research funding to further the next generation of efforts," said Dr. Cleveland. "The Rainwater Prize money will help continue the development of strategies to grow new neurons and suppress Tau chronically."

Rainwater Prize for Innovative Early-Career Scientist:

Dr. Wegmann,Research Group Leader at DZNE,is studying tau protein actions in neurodegenerative diseases by utilizing different experimental models and methodologies to discover tau's normal function and misfunction in the diseased brain. She finds a way to master the difficult translational leap,from test tube to human,by validating biochemical,biophysical and cytological results on postmortem human brain tissue. Her general interest in solving difficult problems drove her to get an engineering degree and later expand into biophysics and brain pathology of tau. Currently,Wegmann and her group focus on identifying new interactions of Tau and determining the role of tau phase separation in Alzheimer's disease and tauopathies.

"I feel very honored! With the prize money,I will further push my research into how the tau protein exerts its toxic effect in different ways,which are not fully understood," said Dr. Wegmann. "We do not understand the actual function of this protein yet and will try to expand our knowledge of its basic biology to aid in disease research."

Drs. Cleveland,Bennett,Miller,and Wegmann were nominated for the Rainwater Prize's Outstanding Innovation ($400,000) and Early-Career ($200,000) Prizes based on published and peer-reviewed research that contributed significantly to neurodegenerative disease research. They were selected based on the quality of research and applicability to a tauopathy,leadership,mentorship,and positive impact within the scientific community.

"I could not be more excited about this year's winners of the Rainwater Prize," said Todd Rainwater,Trustee of the Rainwater Charitable Foundation. "The presentation of the Outstanding Innovation Prize to a collaborative group of scientists advancing drug discovery efforts recognizes the importance of team science. The selection of Dr. Wegmann for this year's early-career prize highlights the exciting scientific discoveries happening in Europe and across the globe. I know my dad would be so proud of these scientists and their important advances to the field."

For more information on this year's prize winners,please visit www.rainwaterprize.org.

About the Rainwater Charitable Foundation's Medical Research

The Rainwater Charitable Foundation (RCF) was created in the early 1990s by renowned private equity investor and philanthropist Richard E. Rainwater. RCF supports a range of programs in K-12 education,medical research,and other worthy causes. In order to deliver on its mission to accelerate the development of new diagnostics and treatments for tau-related neurodegenerative disorders,the Rainwater Charitable Foundation Medical Research Team manages the Tau Consortium and the Rainwater Prize Program. With over $161 million invested in medical research to date,the Rainwater Foundation has helped to advance eight treatments into human trials. For more information,please visit rainwatercharitablefoundation.org.

Corporate Contact:

Jordan Brainerd

Rainwater Charitable Foundation

jbrainerd@rainwatercf.org

(817) 820-2708

Media Contact:

Shannon Fox

Edelman

shannon.fox@edelman.com

(646) 262-7427