The Magic of New Beginnings - News from the Hereditary Disease Foundation

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We’re excited about the beginning of a New Year and the promise 2019 holds for dramatic advances in HD research. Great things are happening…brilliant minds are at work. With your support, we will continue to push until we push HD out of the normal lifespan. Momentum begins with a moment. This is our magical moment…together we will make it happen for our families and for the future.

Read more in our January 2019 Newsletter.

"A new era for treating neurological diseases" HDF 2018 Symposium, Dr. C. Frank Bennett, Ionis

"A new era for treating neurological diseases" HDF 2018 Symposium, Dr. C. Frank Bennett, Ionis

The mood within the room was electric with excitement and hope as Dr. C. Frank Bennett, senior vice president for research and franchise leader for neurological programs at Ionis Pharmaceuticals, took the podium at the Hereditary Disease Foundation October 22, 2018 Gala. He gave a mesmerizing talk

about his work developing antisense therapeutics for neuromuscular and neurological diseases. His pioneering work brought us the world’s first approved treatment for spinal muscular atrophy (SMA). In 2005, Frank started a program to develop antisense therapies for Huntington’s disease. IONIS-HTTrx is designed to treat patients with HD and is the first and only drug to demonstrate reduction of the neurotoxic abnormal huntingtin protein, the underlying cause of HD, in patients. Ionis is partnering with Roche for phase 3 of the trial, which is set to begin in the U.S. and abroad soon!! This is a groundbreaking advance for the field of HD!

Click here to watch in full to Dr. Bennett’s October 22, 2018 Symposium Presentation.

BIG NEWS: Roche/Genentech Announce Site Locations for GENERATION HD1 Trial of RG6042

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Roche/Genentech announce the locations, including 20 sites in the U.S. and 6 in Canada, for their phase 3 study - called GENERATION HD1 - to test efficacy of the huntingtin-lowering therapy RG6042.

This study is open and currently recruiting! This will be a 2-year study in people with early diagnosed HD, involving monthly spinal injections to deliver RG6042 or a placebo, as well as clinical tests and scans to understand the effect of the drug on HD symptoms. Around 660 people age 25-65 will be recruited at approximately 90 sites across the world.

Read the full press release.

"This is a magical moment!" HDF 2018 Symposium - Dr. Shirley Tilghman, Princeton University

“This is a magical moment. This is a moment when discoveries are going to really translate into better medical practice,” said Dr. Shirley Tilghman.

A rapt audience listened intently as Dr. Shirley Tilghman, president emerita and professor of molecular biology and public affairs at Princeton University, spoke about how genes are organized in the genome and regulated during early development.  A member of the National Research Council’s committee that set the blueprint for the United States effort in the Human Genome Project, she was also one of the founding members of the National Advisory Council of the Human Genome Project for the National Institutes of Health.  Dr. Tilghman is a world-renowned leader in the field of molecular biology whose brilliant scientific career as a mammalian development geneticist has advanced our understanding of Huntington’s disease and other disorders.

Click here to watch in full to Dr. Tilghman’s October 22, 2018 Symposium Presentation.

Nancy Wexler visits Ionis Pharmaceuticals to meet the people making the magic happen!

1st Row from left to right- Kristina Bowyer, Leo Sahelijo, Tiffany Baumann, Nancy Wexler, Anne Smith, Holly Kordasiewicz

2nd row left to right- Patrick Cauntay, Chrissa Dwyer (hidden),  Bethany Fitzsimmons, Eric Swayze, Paul Goldberg, Rojer Lane

Eventually C. Frank Bennett, 2018 Leslie Gehry Brenner Prize for Innovation in Science recipient, steps in for a requisite Nancy Wexler hug!

Dr. C. Frank Bennett Receives Leslie Gehry Brenner Prize for Innovation in Science

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We are thrilled to present Frank Bennett with the 2018 Leslie Gehry Brenner Prize for Innovation in Science for his innovative work in bringing us a totally novel class of drugs. We recognize the truly pathbreaking and monumental promise of Frank’s creativity to mold DNA in a way that the toxic HD protein is silenced from mucking up the works in our brain cells. His innovation applies to other brain battering diseases like Alzheimer's, Parkinson's, Lou Gehrig's (ALS) and others. Frank’s novel insight to use the body’s own molecular capacity to create new medicine make him an HD pioneer. His accomplishments honor the spirit and memory of Leslie Gehry by embodying originality, spontaneity, precision and rigor – all critical attributes in a scientist.

Read the Ionis press release.