15 Feb
  • By leilanifera
  • Cause in

TRiNDS

Approach: Neuromuscular focused Contract Research Organization
Funding Impact: Clinical trial support for Biotech/Pharma from a patient-centric perspective

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15 Feb
  • By leilanifera
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Summit

Approach: Utrophin upregulation
Funding Impact: Enabled initiation of Phase 2 clinical trial

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15 Feb
  • By leilanifera
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Sarepta Therapeutics

Approach: Exon skipping
Funding Impact: First drug approved by FDA to treat Duchenne

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15 Feb
  • By leilanifera
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PTC Therapeutics

Approach: Stop codon read-through
Funding Impact: Conditional approval of Translanrna in EU

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15 Feb
  • By leilanifera
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Prosensa/BioMarin

Approach: Exon skipping
Funding Impact: Helped support clinical development of exon skipping drugs
Successful Exit: Prosensa was acquired by BioMarin in 2015

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15 Feb
  • By leilanifera
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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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