Request for Applications: Beyond Exon 51

PPMD announces that it will fund up to $1M in projects by mid-2014 focused on the development of oligos or other techniques to skip exons that are not currently in clinical development, or for multi-exon skipping projects, including the skipping of duplications.

First Round Deadlines and Dates

RFA Released:     August 9th, 2013

Applications Due:    September 13th, 2013

Reviews Due:   November 15th, 2013

Review Responses Due: November 30th, 2013

Funding Start: January 15th, 2014

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Exon-skipping is a technique whereby out-of-frame deletions in a gene can be put back into frame by manipulating splicing patterns to strategically remove an up- or downstream exon in addition to the introns. In Duchenne, the result is a shorter, but often still functional, dystrophin protein. There are currently four companies around the world focused on developing exon-skipping strategies for Duchenne, with antisense oligonucleotides targeting exons 51, 44, 45 and 53 in clinical testing now. As results for the leading exon 51 compounds continue to appear promising, the urgency has increased to expand the technique to correct less common deletions and to develop methods for skipping multiple exons that could also be applied to some duplications as well as deletions.

Starting with a small grant to the laboratories of Steve Wilton and Judith van Deutekom in 2003, PPMD has invested over $600,000 in the development of exon-skipping as a treatment for Duchenne. Now the organization has committed up to $1M in 2014 to capitalize on the momentum created by results for Exon 51 to support preclinical projects focused on

  • exons not currently in clinical development
  • multi-exon skips
  • duplications

Eligibility Criteria 

Applicants should hold an M.D. or Ph.D but can be affiliated either with a research or educational institution or a biotechnology or pharmaceutical company. Applicants may be based within or outside the United States. Program project applications will be considered but a lead applicant must be identified.

Review Criteria

  • Experience and track record of the applicant
  • Availability of additional resources to conduct project
  • Feasibility
  • Relevance to therapy development
  • Impact on the field

Selection Process

PPMD will convene a special ad hoc committee to review all applications received. Written reviews will be supplied to applicants and applicants will be allowed to respond one time to reviewer comments. Based on applicant responses, final funding recommendations will be made by the ad hoc committee.   

Award Requirements

Awards for amounts between $250,000 and $500,000 will be considered. The award period will be one year. All awards will be milestone driven and payments tied to successful completion of milestones. PPMD will not negotiate a return on investment beyond its standard IP policy and will rely upon its standard award letter in lieu of individually negotiated contracts.

Additional Information

Sharon Hesterlee, Ph.D.
Vice President Research


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