15 Feb
  • By leilanifera
  • Cause in

cTAP

Approach: Statistical analysis of natural history studies
Funding Impact: Collaboration amongst biotech/pharma to share data and enrich clinical trial design

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15 Feb
  • By leilanifera
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Nationwide Children’s Hospital

Approach: Exon skipping (Duplication exon 2 mutations)
Funding Impact: Advanced pre-clinical IND package enabling clinical trial for Duchenne boys

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15 Feb
  • By leilanifera
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Exonics

Approach: Gene editing/CRISPR
Funding Impact: Helped found company and fund pre-clinical safety and efficacy research

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01 Nov
  • By leilanifera
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RASRx

Approach: Anti-fibrotic
Funding Impact: Accelerated identification of lead compound and de-risked pre-clinical program

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01 Nov
  • By leilanifera
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MyoTherix

Approach: Anti-inflammatory, anti-osteoporosis, potential steroid sparing therapies
Funding Impact: Industry partnership to sponsor clinical trial for Duchenne

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01 Nov
  • By leilanifera
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Capricor Therapeutics

Approach: Cardiac stem cell derived therapy
Funding Impact: Initiation of Phase 1/2 study (HOPE Clinical Trial)
Successful Exit: Stock Return

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01 Aug
  • By leilanifera
  • Cause in

Bamboo Therapeutics

Approach: Gene therapy for Duchenne
Funding Impact: Accelerated vector manufacturing capabilities
Successful Exit: Acquired by Pfizer in August 2016

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