Pipeline & Progress

Leading with Duchenne muscular dystrophy, our pipeline is rich with personalized treatments targeted to pinpoint and remedy a myriad of genetic mutations in a growing cohort of patients.

Stage 1
Characterize mutation
Stage 2
Prototype and Optimize
Stage 3
Test efficacy
Stage 4
Manufacture and test safety
Stage 5
Clinical Trial
Exon 1 Deletion
Exon 1 Deletion
Exon 20-25 Duplication
Exon 20-25 Duplication
Exon 46-51 Deletion
Exon 46-51 Deletion
Exon 57-59 Deletion
Exon 57-59 Deletion
Exon 8 Duplication
Exon 8 Duplication
Exon 12-20 Duplication
Exon 3-7 Deletion
Exon 33 Deletion
Exon 33 Deletion
Exon 3-7 Deletion
Exon 12-20 Duplication
Exon 7 Duplication
Exon 7 Duplication
Exon 18 Deletion
Exon 18 Deletion
Exon 12-30 Deletion
Exon 12-30 Deletion
Exon 44 Duplication
Exon 44 Duplication
Exon 18-29 Deletion
Exon 18-29 Deletion
Beckers Exon 45-49 Deletion
Beckers Exon 45-49
Deletion
Exon 45-52 Deletion
Exon 45-52 Deletion
Exon 22-39 Deletion
Exon 22-39 Deletion
Exon 45 Deletion
Exon 45 Deletion
Exon 7-8 Deletion
Exon 7-8 Deletion
  • Successful Pre-IND meeting with the FDA for first patient in Fall 2020. 

  • Positive comments received regarding study plan

  • PreIND guidance provides template for all future customized therapeutics

  • NAbs prevent the successful delivery of AAV drugs including CRISPR and micro-dystrophin. Currently estimated at 30-50% of the population and a major hurdle.

  • NAb research study established to measure patient NAb levels. Over 30 samples analyzed thus far with a target of 200. 

  •  Formed NAb technical steering committee to help guide development and implementation of technologies to suppress NAbs in 1-2 years.

  • Human-grade therapeutic in production - expected completion: July 2021

  • Therapeutic constructs under development for several additional patients

  • Targeted dosing of patient 1 is second half of 2022

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