Thalassemia – Market outlook, Epidemiology, Competitive Landscape and Market Forecast Report – 2020 To 2030
Thalassemia is an
inherited blood disorder that reduces the production of functional hemoglobin
(the protein in red blood cells that carries oxygen). This causes a shortage of
red blood cells and low levels of oxygen in the bloodstream, leading to a
variety of health problems.
Etiology-
Mutations in the HBB gene
lead to reduced levels of beta-globin and cause beta-thalassemia. Loss
(deletion) of some or all of the HBA1 and/or HBA2 genes results in a shortage
of alpha-globin, leading to alpha thalassemia.
The competitive
landscape of Thalassemia 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 Thalassemia
across 8 MM market from the centre of Excellence/ Public/ Private hospitals
participated in the study. Insights around current treatment landscape,
epidemiology, clinical characteristics, future treatment paradigm, and Unmet
needs.
Thalassemia
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 ST-400 Sangamo
Therapeutics Phase 1/2
2 VIT-2763 Vifor
(International) Inc. Phase 2
3 Luspatercept Celgene Phase 2
4 CTX001 Vertex
Pharmaceuticals Incorporated Phase 1/2
5 Mitapivat Agios
Pharmaceuticals, Inc. Phase 3
6 Etavopivat tablets Forma
Therapeutics, Inc. Phase 2
7 IONIS TMPRSS6-LRx Ionis
Pharmaceuticals, Inc. Phase 2
8 PTG-300 Protagonist
Therapeutics, Inc. Phase 2
9 Deferasirox Novartis
Pharmaceuticals Phase 2
10 IMR-687 Imara,
Inc. Phase 2
Continued
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