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Boston Children's Hospital — Rare Disease Pipeline | Kisho

Boston Children's Hospital

Other

Orphan Designations

0 approved, 1 designated

FDA Approvals

Active Trials

200 recruiting

Rare Diseases

across 24 areas

News (30d)

Quiet

Boston Children's Hospital is a company with 1 orphan drug designation across 14 rare diseases. Active clinical trials in 30 indications. gene therapy candidates in 1 disease. 32 active policy bills affect their portfolio.

Gene Therapy Active

Competitive Landscape

Diversified Portfolio

Disease-by-disease pipeline progression from orphan designation through clinical trials to FDA approval.

Source: FDA Orphan Drug Designations, ClinicalTrials.gov, Drugs@FDA

Approved: 0

In Trials: 13

Designated Only: 1

14 total diseases

Disease	Drug(s)	Stage
Down syndrome	-	Des.TrialAppr.
Duchenne muscular dystrophy	-	Des.TrialAppr.
Nezelof syndrome	autologous CD34+ bone marrow derived stem cells transduced with a self-inactivating gammaretroviral vector encoding the human IL2RG (yc)	Des.TrialAppr.
cardiovascular disorder	-	Des.TrialAppr.
celiac disease	-	Des.TrialAppr.
congenital heart disease	-	Des.TrialAppr.
cystic fibrosis	-	Des.TrialAppr.
diabetes mellitus	-	Des.TrialAppr.
dystonic disorder	-	Des.TrialAppr.
epilepsy	-	Des.TrialAppr.
multiple sclerosis	-	Des.TrialAppr.
muscular dystrophy	-	Des.TrialAppr.
narcolepsy	-	Des.TrialAppr.
sickle cell disease	-	Des.TrialAppr.