Kisho

University of California San Francisco — Rare Disease Pipeline | Kisho

University of California San Francisco

Other

Orphan Designations

0 approved, 2 designated

FDA Approvals

Active Trials

200 recruiting

Rare Diseases

across 36 areas

News (30d)

Quiet

University of California San Francisco is a company with 2 orphan drug designations across 2 rare diseases. Active clinical trials in 44 indications. gene therapy candidates in 2 diseases. 24 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: 22

Designated Only: 2

24 total diseases

Disease	Drug(s)	Stage
Alzheimer disease	-	Des.TrialAppr.
Parkinson disease	-	Des.TrialAppr.
acute myeloid leukemia	a lentivirally transduced autologous AML vaccine expressing CD80, IL-15, and IL-15Ralpha	Des.TrialAppr.
amyloidosis	-	Des.TrialAppr.
aromatic L-amino acid decarboxylase deficiency	adeno-associated virus type 2 encoding human aromatic L-amino acid decarboxylase	Des.TrialAppr.
brain neoplasm	-	Des.TrialAppr.
breast cancer	-	Des.TrialAppr.
cancer	-	Des.TrialAppr.
cardiovascular disorder	-	Des.TrialAppr.
cervical cancer	-	Des.TrialAppr.
childhood malignant neoplasm	-	Des.TrialAppr.
chronic primary adrenal insufficiency	-	Des.TrialAppr.
congenital heart disease	-	Des.TrialAppr.
dystonic disorder	-	Des.TrialAppr.
epilepsy	-	Des.TrialAppr.
lymphoma	-	Des.TrialAppr.
mitochondrial disease	-	Des.TrialAppr.
mucopolysaccharidosis type 1	-	Des.TrialAppr.
multiple sclerosis	-	Des.TrialAppr.
ovarian cancer	-	Des.TrialAppr.
pulmonary hypertension	-	Des.TrialAppr.
sarcoidosis	-	Des.TrialAppr.
sickle cell disease	-	Des.TrialAppr.
thalassemia	-	Des.TrialAppr.