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AAV-UPF1

Amyotrophic Lateral Sclerosis (ALS)

PreclinicalResearch stage

Key Facts

Indication
Amyotrophic Lateral Sclerosis (ALS)
Phase
Preclinical
Status
Research stage
Company
MeiraGTx

About MeiraGTx

MeiraGTx is focused on advancing a pipeline of gene therapies for serious conditions with high unmet medical need, primarily in ophthalmology and neurology. The company's core strength lies in its integrated platform encompassing vector design, promoter engineering, and manufacturing capabilities. Key strategic partnerships, including a significant collaboration with Janssen Pharmaceuticals, provide validation and resources for its lead programs. The company is navigating the complex clinical and regulatory pathway typical of gene therapy developers.

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Therapeutic Areas

OphthalmologyNeurologyRare Diseases

Other Amyotrophic Lateral Sclerosis (ALS) Drugs

DrugCompanyPhase
Utreloxastat (PTC857)PTC TherapeuticsPhase 2
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RELYVRIO/ALBRIOZA (AMX0035)Amylyx PharmaceuticalsApproved/Commercial
AMX0035Amylyx PharmaceuticalsPhase 3
AMX0114Amylyx PharmaceuticalsPhase 1
Taldefgrobep alfaBiohavenPhase 2/3
AMT-162uniQurePreclinical
TegoprubartEledon PharmaceuticalsPhase 2
Intranasal ForalumabTiziana Life SciencesPhase 2
TDP-43 ProgramKorro BioDiscovery
COYA 302Coya TherapeuticsPhase 2