3
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
3
Active Trials
200 recruiting
6
Rare Diseases
across 10 areas
1
News (30d)
Intellia Therapeutics, Inc. is a company with 3 orphan drug designations across 6 rare diseases. Active clinical trials in 3 indications. 1 news article in the past 30 days.
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 |
|---|---|---|
| ATTRV30M amyloidosis | clustered regularly interspaced short palindromic repeats CRISPR/ Cas9-based gene therapy consisting of a single guide RNA targeting the human TTR gene and a messenger RNA encoding Cas9 | Des.TrialAppr. |
| acute myeloid leukemia | Autologous ex-vivo CRISPR/Cas9 engineered Wilms¿ tumor 1-specific T-cell receptor T cells | Des.TrialAppr. |
| amyloidosis | - | Des.TrialAppr. |
| congenital heart disease | - | Des.TrialAppr. |
| familial amyloid neuropathy | clustered regularly interspaced short palindromic repeats CRISPR/ Cas9-based gene therapy consisting of a single guide RNA targeting the human TTR gene and a messenger RNA encoding Cas9 | Des.TrialAppr. |
| hereditary angioedema | lipid nanoparticle encapsulating single guide RNA (G012267) targeting the human KLKB1 gene and messenger RNA (mRNA000042) encoding Cas9 | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
25
overlap in 2+ diseases
0/6
candidate diseases
1
avg importance: 70
0
affecting portfolio
0% of portfolio targets high unmet need diseases
25
overlap in 2+ diseases
0/6
candidate diseases
1
avg importance: 70
0
affecting portfolio