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New drug for Spinal Muscular Atrophy supported by Emerald BioStructures’structure-bas
02-07-2012, 11:55 AM
Post: #1
New drug for Spinal Muscular Atrophy supported by Emerald BioStructures’structure-bas
Bainbridge Island, WA June 03, 2011. Another drug candidate supported by structural insight from Emerald BioStructures has reached clinical trials. The US Food and Drug Administration has granted approval to commence a Phase I safety study for RG3039, formerly called D157495, an investigational new drug (IND) being developed to treat Spinal Muscular Atrophy (SMA).

The RG3039 IND is the third drug candidate based on Emerald insights to be announced in recent months and eighth IND overall for Emerald’s clients. No structural biology CRO has a more significant track record in bringing drugs to clinic. “Structure-based insights are key to the rapid development of therapeutic agents,” said Lance Stewart, Ph.D., CEO of Emerald BioStructures.

The discovery of RG3039 by Families of Spinal Muscular Atrophy (SMA) was aided by Emerald’s three-dimensional structures of C5-substituted Quinazolines bound to DcpS, an enzyme involved in gene expression.This work is documented in the peer-reviewed paper, “DcpS as a therapeutic target for spinal muscular atrophy.”

Structure based drug design at Emerald

Emerald BioStructures is a leading Contract Research Organization (CRO) dedicated to determining three-dimensional structures of macromolecules that have the potential to become important drug targets. Families of Spinal Muscular Atrophy (SMA) used the three-dimensional structures discovered at Emerald as the basis of this novel drug, which has gone to clinic with development partner RepliGen. “Emerald’s structural biology services informed our target assessment and helped us guide lead optimization strategies,” said Jill Jarecki, Ph.D., Research Director of Families of SMA. “This approach helped us efficiently determine the druggability of our targets.”

First-ever drug to treat Spinal Muscular Atrophy (SMA)

SMA is a genetic disorder affecting about 1 in 6,000 births per year and is the leading genetic cause of death in children under the age of two.SMA is caused by deletion or mutation of both copies of the SMN1 gene, which produces an essential protein known as SMN.

Emerald’s work contributed to the discovery of a binding molecule, DcpS, that represents a novel therapeutic target for modulating gene expression. Emerald helped to show that the inhibition of DcpS can negate the harmful effects of the missing or mutated SMN1 gene.

About Emerald BioStructures

Emerald BioStructures is an integrated gene-to-structure contract research organization that provides collaborative drug discovery services to pharmaceutical companies, biotechnology companies, academic institutions, and government facilities. The company operates a high-throughput platform leveraged for fragment-based lead discovery and structure-based drug design. Emerald’s work provides a solid foundation for the discovery of highly selective, efficacious drugs.

About Families of Spinal Muscular Atrophy

Families of SMA is a non-profit 501©3 tax exempt organization with 30 Chapters throughout the United States and over 70,000 members and supporters. Families of SMA funds and directs the leading SMA research programs. The successful results and progress from basic research to drug discovery programs to clinical trials provide real hope for families and patients.
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