Vutrisiran
- Generic Name
- Vutrisiran
- Brand Names
- Amvuttra
- Drug Type
- Biotech
- Chemical Formula
- -
- CAS Number
- 1867157-35-4
- Unique Ingredient Identifier
- GB4I2JI8UI
- Background
Vutrisiran is a double-stranded small interfering ribonucleic acid (siRNA) that targets wild-type and mutant transthyretin (TTR) messenger RNA (mRNA). This siRNA therapeutic is indicated for the treatment of neuropathies associated with hereditary transthyretin-mediated amyloidosis (ATTR), a condition caused by mutations in the TTR gene. More than 130 TTR mu...
- Indication
Vutrisiran is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
- Associated Conditions
- Polyneuropathies
- Associated Therapies
- -
A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
- First Posted Date
- 2024-11-08
- Last Posted Date
- 2024-12-17
- Lead Sponsor
- Alnylam Pharmaceuticals
- Target Recruit Count
- 800
- Registration Number
- NCT06679946
- Locations
- 🇬🇧
Clinical Trial Site, Manchester, United Kingdom
HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
- Conditions
- Interventions
- First Posted Date
- 2019-11-06
- Last Posted Date
- 2024-12-10
- Lead Sponsor
- Alnylam Pharmaceuticals
- Target Recruit Count
- 655
- Registration Number
- NCT04153149
- Locations
- 🇬🇧
Clinical Trial Site, Middlesbrough, United Kingdom
HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
- Conditions
- Interventions
- First Posted Date
- 2018-11-30
- Last Posted Date
- 2024-11-14
- Lead Sponsor
- Alnylam Pharmaceuticals
- Target Recruit Count
- 164
- Registration Number
- NCT03759379
- Locations
- 🇬🇧
Clinical Trial Site, London, United Kingdom
Drug Development Updates
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