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Therapeutic Area

Enzyme Replacement Therapy

Therapeutic area intelligence for Enzyme Replacement Therapy with 4 indexed intelligence signals across regulatory, clinical, competitive, and market developments.

Signals
4
Catalysts
0
Companies
1

Companies

Anagram Therapeutics →

Assets

ACP-501 →
ANG003 →
NULIBRY →

Recent signals(4)

RegulatoryAugust 10, 2026

FDA Accepts Supplement Application for NULIBRY by Sentyln Therapies

The FDA has accepted the supplemental application NDA214018 for NULIBRY, an enzyme replacement therapy containing fosdenopterin hydrobromide, submitted by Sentyln Therapies.

RegulatoryJuly 20, 2026

FDA Accepts Supplemental NDA for NULIBRY by Sentyln Therapies

The FDA has accepted a supplemental NDA for NULIBRY, an enzyme replacement therapy for a rare disease.

CompetitiveJuly 10, 2026

Anagram Therapeutics initiates Phase 2 trial for ANG003 in exocrine pancreatic insufficiency

Anagram Therapeutics is conducting Phase 2 study to evaluate the safety and efficacy of ANG003 for exocrine pancreatic insufficiency.

ClinicalJune 26, 2026

First-in-Human Trial of ACP-501 for Familial LCAT Deficiency Initiated

A clinical trial has been initiated to evaluate the safety and efficacy of ACP-501, a recombinant human LCAT, in a patient with familial LCAT deficiency.

Upcoming catalysts

No catalysts indexed for this entity yet.

Indications

Genetic Disorders →
Rare Disease →
Rare Diseases →

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