"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.

Update from Roche/Genentech on RG6042 Huntington’s disease global development program: Two clinical studies to begin by end of 2018


Over the past months we and our partner Ionis Pharmaceuticals have been heavily engaged with communities around the world (patient groups, medical professionals, Health Authorities and payers) to collaborate and build the RG6042 global development programme and upcoming studies. We are eager for RG6042 to advance into further clinical development. In addition, as announced last month, the European Medicines Agency granted RG6042 PRIME (“PRIority MEdicine”) designation, which provides promising medicines enhanced interactions with the agency and the potential for accelerated evaluation.


Exciting news from Ionis Pharmaceuticals! Potentially faster approval for HTTRx!

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Ionis Pharmaceuticals, Inc. announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to IONIS-HTTRx for the treatment of people with Huntington's disease (HD). This PRIME designation means this potentially disease-modifying drug for people with Huntington's disease to regulatory approval faster. This is exciting news! Read the full press release.