Cure SMA’s Seed Funding Yields New Investments in Research

By Cure SMA | Published On February 22, 2016

Cure SMA’s Seed Funding Yields New Investments in Research

No single group can develop a treatment for SMA alone—it requires collaboration between academics, industry, government, and families. Cure SMA brings all of those groups together, breaking down roadblocks that might delay important developments.

Cure SMA provides early-stage funding for promising new ideas and novel treatment approaches. In doing so, we lower the risk and attract larger investments from industry, government, and other funding sources as drug candidates move through the process.

It can cost upwards of $1 billion to bring a therapy through the development process to FDA approval. In addition, we are working to increase the number of new therapies in the SMA pipeline, to insure we have treatments for all ages and stages of SMA. Attracting investment from government, pharmaceutical companies, and other organizations provides the resources necessary to accomplish these goals.

Here are a few of the recent successes we’ve had in leveraging early investments.

Gene Therapy

Through a series of grants, Cure SMA invested $845,000 in two different gene therapy approaches. In 2013, the National Institute of Neurological Disorders and Stroke (NINDS) awarded Dr. Brian Kaspar a $4 million grant, based on data generated with Cure SMA funding.

The technology for this gene therapy was then licensed to a company for clinical trials. Earlier this month, AveXis held an initial public offering (IPO), raising over $95 million from investors to move gene therapy into Phase 2 clinical trials.

Antisense Oligonucleotides (ASOs)

An ASO is a small snippet of synthetic genetic material used to modulate gene expression. In 2013, we made a $150,000 grant to Dr. Chris Lorson and Dr. Arthur Burghes to help optimize an ASO that binds to a novel sequence called E1. The E1 ASO could be used to improve the function of SMN2, the SMA “backup gene.”

Last year, Dr. Lorson announced that they were able to optimize the E1 directed ASO that increased life span in severe mouse models of SMA by four times and in intermediate mouse models of SMA by seven times. Based on that data, Dr. Lorson has recently received two additional grants, totaling $550,000, from MDA, GSF, and FightSMA, to continue moving this compound forward.

Muscle Activators

In 2013, Cure SMA made a grant of $100,000 to Dr. Jeff Jasper at Cytokinetics. This grant was intended to support the development of a small molecule that protects and activates skeletal muscles that typically weaken in SMA.

In 2014, Astellas Pharmaceuticals announced a partnership with Cytokinetics to bring this drug class (specifically CK-2127107) to clinical trials for SMA. CK-2127107 is currently being tested in Phase 2 clinical trials in teens and adults with SMA, and Astellas has invested $55 million in order to move the drug to this point.

Topics: Our Impact, Research, Front Page News

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