Brandeis and Brigham and Women's Hospital license technology for Gaucher's to Amicus Therapeutics

Waltham, MABrandeis University and Brigham and Women's Hospital today agreed to grant a license option to Amicus Therapeutics for a jointly-developed novel pharmaceutical technology that could be instrumental in finding new treatments for Gaucher Disease. Biochemists Raquel Lieberman, Gregory Petsko and Dagmar Ringe, each affiliated with both Brandeis and Brigham and Women's, invented a patentable technology related to the structure of acid beta-glucosidase, also known as GCase, and methods for identifying therapeutic agents. In collaboration with scientists at Amicus Therapeutics, the inventors discovered that GCase, the protein that is mutated in Gaucher Disease, undergoes a structural change near its active site upon binding to a small molecule that is in clinical trials for the treatment of the disease.

One emerging type of treatment for genetic diseases such as Gaucher Disease involves the use of small molecules known as pharmacological chaperones, which are proposed to help mutated, less stable proteins perform their normal function in the cells of patients with the disease. The identification of appropriate pharmacological chaperones and understanding how they work in the body are aided by knowing the structure of the target protein under different conditions and when bound to the small molecule.

Cranbury, New Jersey-based Amicus Therapeutics, which specializes in the discovery and development of pharmacological chaperones, will use this structure of GCase to look for additional small molecules that can keep the protein properly folded and stabilized.

"This work helps explain the way pharmacological chaperones work to stabilize proteins that are rendered less stable by genetic mutations in diseases such as Gaucher, Anderson-Fabry, and other genetic disorders," explained Petsko, the Gyula and Katica Tauber Professor of Biochemistry and Chemistry, Brandeis University and Adjunct Professor, Department of Neurology and Center

Contact: Laura Gardner
Brandeis University

Page: 1 2

Related biology news :

1. New Brandeis research sheds light on memory by erasing it
2. National Academy of Sciences elects 2 Brandeis University scientists to membership
3. Biogen Idec acquires Brandeis spin-out
4. HHMI awards Brandeis professor $1 million to draw minorities into science
5. Carl and Ruth Shapiro family donates $25 million for new science center at Brandeis University
6. American Physical Society honors outstanding achievements of two Brandeis scientists
7. Brandeis biologist wins top award in neuroscience
8. Brandeis wins $1 million grant to train scientists in solving 21-century biomedical problems
9. AAAS and Brandeis University announce 2005 Fellows
10. Brandeis leads collaborative team in researching chemical processes in brain
11. MIT, Brigham: Nanoparticles armed to combat cancer

Post Your Comments:

(Date:10/9/2015)... Oct. 09 2015 ... of the "Samsung Galaxy S6 Fingerprint ... to their offering. --> http://www.researchandmarkets.com/research/6g9l3b/samsung_galaxy_s6 ... "Samsung Galaxy S6 Fingerprint Sensor - ... offering. --> Research and Markets ...
(Date:10/8/2015)... , Oct. 8, 2015 Synaptics ... human interface solutions, announced today that it will ... fiscal 2016 on Thursday, October 22, 2015, after ... a corresponding conference call for analysts and investors ... which management may discuss forward-looking information.    ...
(Date:10/8/2015)... CITY , 8. Oktober 2015 /PRNewswire/ ... ein global tätiges Unternehmen des Bereiches Tracking, ... Vertrag mit der Gefängnisbehörde Virginias (Department of ... Rahmen elektronische Überwachungsdienste für alle Strafen geliefert ... Derek Cassell , Präsident für den Amerikanischen ...
Breaking Biology News(10 mins):
(Date:10/9/2015)... , Oct. 9, 2015 On October ... the Congressional Record her statement recognizing the third annual ... 11-17. IPAW is sponsored by the Plasma Protein ... is designed to:   , Raise global awareness ... plasma donors in saving and improving lives , ...
(Date:10/9/2015)... Governor Tom Wolf announced today ... on developing T-cell receptor cancer immunotherapy treatments, will create ... expansion project. Pennsylvania , and ... 110 new, high-paying jobs will be created with this ... proposal includes sustained funding for the life science sector ...
(Date:10/9/2015)... HERSHEY, Pa. and ROCKVILLE, ... Therapeutics, Inc. ("ITI"), a privately-held biotechnology company developing ... today announced that it has entered into an ... on LAMP-vax products for the treatment or prevention ... Upon execution of the agreement, Immunomic Therapeutics will ...
(Date:10/9/2015)... , Oct. 9, 2015  DePuy Synthes Trauma* announced ... with Q-PACK ®  Technology**, the only pre-hydrated demineralized ... extremity procedures (foot and ankle, hand and wrist), including ... made here at the 2015 Orthopaedic Trauma Association (OTA) ... natural scaffold for new bone growth) and osteoinductive 2,3 ...
Breaking Biology Technology:
Cached News: