VivoSeq
Rare Disease

Hereditary Transthyretin Amyloidosis

7

Pipeline Programs↗

1

Active Trials↗

5

Companies Involved↗

Hereditary Transthyretin Amyloidosis is an early-stage landscape with 7 programs across 5 companies. The most active players are Intellia Therapeutics, Akcea Therapeutics, Alnylam Pharmaceuticals. 1 therapy is approved with 4 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

32/100

Pipeline Velocity

6/100

Mech. Concentration

25/100

Completion Rate

100/100

Attrition Rate

0/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

Intellia Therapeutics logo

Intellia Therapeutics

1 program

Phase 1A

Akcea Therapeutics

1 program

Phase 3Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals

4 programs

ApprovedK

K-Group Alpha, Inc., a wholly owned subsidiary of Zentalis Pharmaceuticals, Inc.

1 program

PreclinicalPfizer logo

Pfizer

0 programs

Preclinical

Pipeline

Programs by Phase

Analysis

Modality Breakdown

83%

RNA therapy

5 of 6 classified programs — the dominant modality here.

17%ALL OTHERSSmall molecule117%

RNA therapy leads; the ring shows all 6 classified programs, each share of the total. Modality classified for 100% of 7 pipeline programs.

Analysis

Trial Status

5

clinical trials

Completed4 · 80%
Recruiting1 · 20%

Clinical Trials

Trial Activity

HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
Completed·Alnylam Pharmaceuticals logoAlnylam Pharmaceuticals·Phase 3·NCT03759379·164 enrolled
Retrospective Study Collecting Neurological Follow-up of Hereditary Transthyretin Amyloidosis (ATTRv) Patients Included in B3461028 and B3461045.
Completed·Pfizer logoPfizer·N/A·NCT05560555·5 enrolled
Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)
Completed·Intellia Therapeutics logoIntellia Therapeutics·Phase 1·NCT04601051·72 enrolled
A Study to Examine the Clinical Effectiveness of Tafamidis in Patients With Mixed Phenotype Hereditary Transthyretin Amyloidosis
Completed·Pfizer logoPfizer·N/A·NCT05139680·10 enrolled
TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy
Recruiting·Alnylam Pharmaceuticals logoAlnylam Pharmaceuticals·Phase 3·NCT07223203·125 enrolled

Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)