2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
2
Active Trials
200 recruiting
2
Rare Diseases
across 5 areas
1
News (30d)
Beam Therapeutics is a company with 2 orphan drug designations across 2 rare diseases. Active clinical trials in 2 indications. gene therapy candidates in 1 disease. 19 active policy bills affect their portfolio. 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 |
|---|---|---|
| alpha 1-antitrypsin deficiency | a lipid nanoparticle-based therapy consisting of a guide RNA that targets the SERPINA1-c.1096G>A allele and a messenger RNA that encodes an ABE protein | Des.TrialAppr. |
| sickle cell disease | autologous CD34+ cell therapy comprised of hematopoietic stem and progenitor cells base edited with one guide RNA and a single messenger RNA encoding an adenine base editor | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
1/2
candidate diseases
1
avg importance: 40
19
affecting portfolio
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
1/2
candidate diseases
1
avg importance: 40
19
affecting portfolio