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RGDI Promotes Drug Discovery

Publication date: 21 Apr 2019

With effective therapies for rare diseases being hard to come by, how can drug development be accelerated?  Better collaborative relationships among patients, researchers, medical professionals and commercial partners may be the key.  Rare Genomics Institute is seeking to leverage its resources to help bring about such collaborations. A specialized project team, the Rare Genomics Drug Initiative (RGDI), has been formed to promote the development of new drugs and therapeutic approaches to benefit rare disease patients.  The team aims to advance the discovery of drugs for rare disorders with limited therapeutic options by addressing patient needs with a strong foundation of knowledge and strategic partnerships.

RGDI is open to exploring all types of therapeutic modalities.  Small molecules, biologics, genetic therapies and cellular treatments are all potential avenues for treatments.  The primary goal is to bridge the gap between patients with rare disorders and the research community seeking scientific understanding and innovative solutions.  Additionally, RGDI seeks to bring together nonprofit groups and the pharmaceutical industry to identify and find solutions for existing treatment gaps.

Building a network of researchers who are at the forefront of understanding rare disease biology will speed the identification of novel treatments.  Increasingly, the discovery and understanding of genetic and systemic pathways involved in the development of rare disorders are opening doors to previously unknown therapeutic strategies.  On the other hand, “old” approaches need not be abandoned. The repurposing of existing drugs for new indications can be a highly effective strategy. Expanded usage of existing drugs can drive pharmaceutical company support.  Safety characteristics of a marketed drug may already be well known, reducing the time of the clinical trial process, expediting regulatory approval and speeding the time to market.

RGDI also looks to encourage interactions between companies and the clinical community to find patients who can benefit from new treatments.  Further, it seeks to align non-profit patient advocacy groups, academic researchers, physicians and the biotech industry to create a complementary team to provide hope for patients with devastating conditions and unmet clinical needs.

The RGDI team aims to bring new therapeutic options to rare disease patients by focusing the efforts of multiple stakeholders to a common purpose.  Building partnerships between those with the scientific means and those with medical needs is RGDI’s main strategy. We invite anyone interested in becoming involved with this initiative to contact the RGDI team at