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