12 Apr
  • By Debra Miller
  • Cause in

CureDuchenne Shares Pfizer’s Update Regarding Initiation of Gene Therapy Trial

Today, Pfizer announced that the first patient was dosed in their mini-dystrophin gene therapy trial.  This is an exciting day for the Duchenne community.  Pfizer has the experience to conduct this trial, and manufacturing capacity to see it through to success.  Of course, this is an early stage safety trial and there is a long […]

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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
  • Cause in

Summit

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

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15 Feb
  • By leilanifera
  • Cause in

Sarepta Therapeutics

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

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15 Feb
  • By leilanifera
  • Cause in

PTC Therapeutics

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

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15 Feb
  • By leilanifera
  • Cause in

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
  • 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
  • Cause in

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
  • Cause in

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
  • Cause in

RASRx

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

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