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Rare Diseases And Orphan Drugs: New Partnership And Other Happenings

May 9, 2013

The following discusses a new partnership and other happenings in the area of rare diseases and orphan drug development :

I – Nimbus Discovery And Shire

Nimbus Discovery LLC, a biotechnology startup company in Cambridge, Massachusetts, announces May 8, 2013, an agreement with Shire to co-develop first-in-class small-molecule therapies for rare genetic diseases known as Lysosomal Storage Disorders (LSDs). Nimbus Discovery is backed by life science investors, including Atlas Venture, SR One, Lilly Ventures, and Bill Gates.

Under the agreement :

•   Nimbus Discovery will use its research and development platform to profile molecules against the agreed upon disease target
•   Nimbus Discovery will then deliver a drug candidate that is ready to enter late preclinical studies
•   Nimbus Discovery controls and conducts all research up to the achievement of drug candidate status
•   Shire will have an exclusive option to acquire the program
•   If Shire acquires the program, then Shire will be responsible for the drug candidate’s clinical development & commercialization
•   Nimbus Discovery is eligible to receive preclinical, development, and commercial stage milestones.

The purpose of the collaboration is to “utilize the Nimbus breakthrough computational chemistry approach to discover and develop novel, disease-altering therapies … the significant potential of this partnership is the development of the first small molecule agents designed to penetrate inaccessible tissues while offering the convenience of an orally administered pill.”

LSDs includes about 50 or more inherited diseases. LSDs are diseases where missing enzymes cause buildups of certain complex carbohydrates in the lysosomes of cells that can lead to organ damage and failure. Shire’s Human Genetic Therapies (HGT) unit currently provides Enzyme-Replacement Therapies (ERTs) for LSDs such as Hunter Syndrome and Fabry Disease.

II – NORD Funding Opportunities

The National Organization for Rare Disorders (NORD) is offering 7 research grant awards in 2013:

•   Adult Primary Lateral Sclerosis (PLS)
•   Dubowitz Syndrome (DS)
•   Glycine Encephalopathy, (Nonketotic Hyperglycinemia) (NKH)
•   Growth Failure in Children with Cardifaciocutaneous Syndrome (CFC)
•   Post-Orgasmic Illness Syndrome (POIS)
•   Pseudomyxoma Peritonei (PMP) — (two grants).

By May 15, 2013 a letter of intent is due. NORD will send invitations to submit a full proposal in early July and these full proposals will be due by September 11, 2013.

III – A Call For Abstracts

A previous Orphan Druganaut Blog Post, presents an announcement from the U.S. Agency for Healthcare Research and Quality (AHRQ) that it will sponsor a special journal supplement for the Journal of General Internal Medicine (JGIM), on research methods for evaluating health outcomes in rare diseases.

The announcement invites authors to submit abstracts for manuscripts that describe original research or systematic reviews on methodologies for studying patient-centered health outcomes in rare diseases. The submissions should show how the authors’ approach applies to problems encountered in the design, analysis, or conduct of rare disease research. The following are key dates for the Abstract submission :

•   05/31/13: Abstract submission forms are due
•   06/26/13: Abstract authors who are selected to write a full manuscript are notified
•   11/22/13: Complete manuscripts are due
•   2014: Publication of the supplement to the Journal of General Internal Medicine.

Please reference AHRQ announcement and Abstract information for additional details.

IV – Harvard Biomedical Research Accelerator

Harvard announces on April 30, 2013, that the university receives a $50 million gift from the Blavatnik Family Foundation for the purpose to “expedite the development of basic science discoveries into new breakthrough therapies for patients and cures for disease”, in an initiative called the Blavatnik Biomedical Accelerator. The Accelerator will identify promising research, discoveries, and technology coming out of Harvard and help them in the development stages of new therapies so that they can quickly be brought to market. It will be interesting to follow and observe whether therapies for rare diseases are pursued as part of this accelerator.

Please Note: “Global Thinking” by Benjamin D. Esham [Public domain] | Wikimedia Commons.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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