1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
1
Active Trials
200 recruiting
4
Rare Diseases
across 11 areas
0
News (30d)
Quiet
EpiCrispr Biotechnologies, Inc. is a company with 1 orphan drug designation across 4 rare diseases. Active clinical trials in 1 indication. 1 active policy bill affects their portfolio.
Disease-by-disease pipeline progression from orphan designation through clinical trials to FDA approval.
Source: FDA Orphan Drug Designations, ClinicalTrials.gov, Drugs@FDA
| Disease | Drug(s) | Stage |
|---|---|---|
| malignant carotid body paraganglioma | Adeno-Associated Viral Vector Expressing Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/dCas-based Gene Therapy consisting of a Single Guide RNA targeting the Human D4Z4 Locus and a messenger RNA encoding dCasONYX | Des.TrialAppr. |
| muscular dystrophy | - | Des.TrialAppr. |
| post-treatment Lyme disease syndrome | Adeno-Associated Viral Vector Expressing Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/dCas-based Gene Therapy consisting of a Single Guide RNA targeting the Human D4Z4 Locus and a messenger RNA encoding dCasONYX | Des.TrialAppr. |
| treatment-refractory schizophrenia | Adeno-Associated Viral Vector Expressing Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/dCas-based Gene Therapy consisting of a Single Guide RNA targeting the Human D4Z4 Locus and a messenger RNA encoding dCasONYX | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
83
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
1
affecting portfolio
0% of portfolio targets high unmet need diseases
83
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
1
affecting portfolio