The Winter 2019 issue of Compass in Now Available Online

The Winter 2019 issue of Compass is now available online. This issue covers Cure SMA’s latest drug discovery grant funding and features an updated SMA drug pipeline.

Drug discovery, also called translational research, is the second step in the drug development process. Drug discovery takes the seed ideas discovered in basic research, the first stage, and converts them into usable drugs that can then be tested in the third stage, clinical trials.

Our unique approach minimizes the most common challenges of drug discovery by building a diverse “pipeline” of drug candidates.

  • We invest in a number of projects at a time, which means that if a drug candidate fails, several others can take its place.
  • We fund projects that represent a variety of therapeutic approaches, attacking SMA from all sides.
  • We provide seed funding for new projects. Traditionally, it has been difficult to get pharmaceutical companies to invest in research for rare diseases like SMA. By providing early stage funding, we lower the risk and attract larger investments from industry and government as drug candidates move through the process.
  • We are unbiased as we evaluate all possible treatment opportunities, and we prioritize, select, and manage our drug discovery projects through a Translational Advisory Committee, comprised of industry experts.

This current round of funding reflects this diverse, unbiased strategy. The grants given represent the two of the most significant and promising avenues for SMA treatment.

For More Information

Check out the grant announcements in our news section for additional information about our grantees:

$300,000 to Umrao Monani, PhD

$300,000 to Kevin Hodgetts, PhD

These grants were generously funded by a donation made to Cure SMA anonymously in honor of William N. Kanehann. We are grateful for this amazing donation in memory of Billy’s life.

To read past issues of Compass, visit our research publications page.

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