Kisho

University of Alabama at Birmingham — Rare Disease Pipeline | Kisho

University of Alabama at Birmingham

Other

Orphan Designations

0 approved, 1 designated

FDA Approvals

Active Trials

200 recruiting

Rare Diseases

across 40 areas

News (30d)

Quiet

University of Alabama at Birmingham is a company with 1 orphan drug designation across 22 rare diseases. Active clinical trials in 42 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: 21

Designated Only: 1

22 total diseases

Disease	Drug(s)	Stage
Alzheimer disease	-	Des.TrialAppr.
Down syndrome	-	Des.TrialAppr.
Parkinson disease	-	Des.TrialAppr.
amyloidosis	-	Des.TrialAppr.
amyotrophic lateral sclerosis	-	Des.TrialAppr.
breast cancer	-	Des.TrialAppr.
cancer	-	Des.TrialAppr.
cardiovascular disorder	-	Des.TrialAppr.
cervical cancer	-	Des.TrialAppr.
childhood malignant neoplasm	-	Des.TrialAppr.
congenital heart disease	-	Des.TrialAppr.
cystic fibrosis	-	Des.TrialAppr.
diabetes mellitus	-	Des.TrialAppr.
epilepsy	-	Des.TrialAppr.
malignant glioma	Oncolytic Herpes Simplex Virus Expressing IL-12 - M032	Des.TrialAppr.
multiple sclerosis	-	Des.TrialAppr.
neurofibromatosis	-	Des.TrialAppr.
neurofibromatosis type 1	-	Des.TrialAppr.
ovarian cancer	-	Des.TrialAppr.
pulmonary hypertension	-	Des.TrialAppr.
sarcoma	-	Des.TrialAppr.
sickle cell disease	-	Des.TrialAppr.