3
Orphan Designations
0 approved, 3 designated
0
FDA Approvals
0
Active Trials
5
Rare Diseases
across 7 areas
0
News (30d)
Quiet
Beijing Genecradle Therapeutics Co., Ltd. is a company with 3 orphan drug designations across 5 rare diseases. gene therapy candidates in 1 disease.
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 |
|---|---|---|
| Wilson disease | recombinant adeno-associated virus serotype 5 vector carrying the codon-optimized and truncated human ATP7B gene | Des.TrialAppr. |
| glycogen storage disease II | recombinant adeno-associated virus serotype 9 vector expressing codon optimized human GAA gene | Des.TrialAppr. |
| spinal muscular atrophy with congenital bone fractures 1 | self-complementary recombinant adeno-associated viral vector (scAAV) containing a single-stranded transgene encoding a codon-optimized human SMN1 gene | Des.TrialAppr. |
| spinal muscular atrophy with congenital bone fractures 2 | self-complementary recombinant adeno-associated viral vector (scAAV) containing a single-stranded transgene encoding a codon-optimized human SMN1 gene | Des.TrialAppr. |
| spinal muscular atrophy, type IV | self-complementary recombinant adeno-associated viral vector (scAAV) containing a single-stranded transgene encoding a codon-optimized human SMN1 gene | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
8
overlap in 2+ diseases
1/5
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
8
overlap in 2+ diseases
1/5
candidate diseases
0
avg importance: 0
0
affecting portfolio