hLB-001

Alexion Pharmaceuticals, Inc. · Phase 2 active Small molecule

At a glance

Generic name	hLB-001
Sponsor	Alexion Pharmaceuticals, Inc.
Modality	Small molecule
Phase	Phase 2

Approved indications

No approved indications tracked.

Common side effects

No common side effects on file.

Key clinical trials

Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy
Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia (PHASE1, PHASE2)

Primary sources

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Source	Used for
ClinicalTrials.gov	Trial enrolment, design, endpoints, results

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

hLB-001 CI brief — competitive landscape report
hLB-001 updates RSS · CI watch RSS
Alexion Pharmaceuticals, Inc. portfolio CI