2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
4
Active Trials
200 recruiting
7
Rare Diseases
across 13 areas
0
News (30d)
Quiet
Tenaya Therapeutics is a company with 2 orphan drug designations across 7 rare diseases. Active clinical trials in 4 indications. 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 |
|---|---|---|
| congenital heart disease | - | Des.TrialAppr. |
| hypertrophic cardiomyopathy | a native AAV9 capsid with a genomic cassette containing a cardiomyocyte-specific promoter and the wild-type human MYBPC3 gene | Des.TrialAppr. |
| hypertrophic cardiomyopathy 1 | a native AAV9 capsid with a genomic cassette containing a cardiomyocyte-specific promoter and the wild-type human MYBPC3 gene | Des.TrialAppr. |
| hypertrophic cardiomyopathy 9 | a native AAV9 capsid with a genomic cassette containing a cardiomyocyte-specific promoter and the wild-type human MYBPC3 gene | Des.TrialAppr. |
| malignant carotid body paraganglioma | A native AAV9 capsid with a genomic cassette containing a cardiomyocyte-specific promoter and the wild-type human PKP2 gene | Des.TrialAppr. |
| post-treatment Lyme disease syndrome | A native AAV9 capsid with a genomic cassette containing a cardiomyocyte-specific promoter and the wild-type human PKP2 gene | Des.TrialAppr. |
| treatment-refractory schizophrenia | A native AAV9 capsid with a genomic cassette containing a cardiomyocyte-specific promoter and the wild-type human PKP2 gene | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
83
overlap in 2+ diseases
0/7
candidate diseases
0
avg importance: 0
1
affecting portfolio
0% of portfolio targets high unmet need diseases
83
overlap in 2+ diseases
0/7
candidate diseases
0
avg importance: 0
1
affecting portfolio