Indication name: Leber
Congenital Amaurosis (LCA)
Leber Congenital Amaurosis (LCA)
can result from mutations in at least 14 genes, all of which are necessary for
normal vision. Mutations in the CEP290, CRB1, GUCY2D, and RPE65 genes are the
most common causes of the disorder, while mutations in the other genes
generally account for a smaller percentage of cases. In rare cases, LCA is
inherited as an autosomal dominant genetic disorder. Mutations in three genes,
CRX, IMPDH1, and OTX2 are currently known to be associated with this type of
LCA. Thelansis Epidemiology study reflects incidence of Leber congenital
amaurosis occurs in 2 to 3 per 100,000 newborns. It is one of the most common
causes of blindness in children.
Competitive landscape of LCA
includes country specific approved as well as pipeline therapies. Any asset/
product specific designation or review and Accelerated Approval are being
tracked and supplemented with analyst commentary.
KOLs
insights of LCA across 8 MM market from center of Excellence/ Public/
Private hospitals participated in the study. Insights around current treatment
landscape, epidemiology, clinical characteristics, future treatment paradigm
and Unmet needs.
LCA Market Forecast: Patient
Based Forecast Model (MS. Excel Based Automated Dashboard) which Data Inputs
with sourcing , Market Event and Product Event , Country specific Forecast
Model , Market uptake and patient share uptake , Attribute Analysis , Analog
Analysis , Disease burden and pricing scenario, Summary and Insights.
S. No Asset Company Stage
1 sepofarsen ProQR Therapeutics Phase 3
2 SAR439483 Atsena Therapeutics Inc. Phase 2
3 rAAV2-CB-hRPE65 Applied Genetic Technologies Corp Phase 2
4 voretigene neparvovec-rzyl Spark Therapeutics Phase 2
5 AAV RPE65 MeiraGTx UK II Ltd Phase 2
6 QLT091001 QLT Inc. Phase 1
7 (HuRPE) cells Eyecure Therapeutics Inc. Early Phase 1
8
EDIT-101 Editas Medicine,
Inc. Phase 2
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