Delving into the Latest Updates on Vesemnogene Lantuparvovec with Synapse

Overview

Basic Info

Drug Type

AAV based gene therapy

Synonyms

AAV-hSMN1

Target

SMN1

Action

modulators

Mechanism

survival of motor neuron 1, telomeric modulators

Therapeutic Areas

Nervous System DiseasesOther Diseases

Active Indication

Spinal Muscular Atrophy

Inactive Indication-

Originator Organization

Lantu Biopharma Inc.

Active Organization

Lantu Biopharma Inc.

Inactive Organization-

License Organization-

Drug Highest PhasePhase 3

First Approval Date-

Regulation-

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The study objective is to determine the real-world safety and effectiveness of Vesemnogene lantuparvovec for the treatment of SMA.
The specific objectives are:
* To determine clinical effectiveness of Vesemnogene lantuparvovec therapy for SMA as evaluated by developmental gross motor milestone and survival.
* To describe the safety profile of Vesemnogene therapy for SMA as evaluated by adverse events reporting and laboratory tests, and monitoring of Adverse events of special interest.

Start Date15 Oct 2025

Sponsor / Collaborator

Lantu Biopharma Inc.

NCT06288230

/ RecruitingPhase 1/2

Study of AAV-hSMN1 (Vesemnogene Lantuparvovec) Gene Therapy in Subjects With Progressive Spinal Muscular Atrophy

This is an interventional study to evaluate safety and efficacy of AAV-hSMN1 in spinal muscular atrophy patients.

Start Date20 Oct 2024

Sponsor / Collaborator

Lantu Biopharma Inc.

100 Clinical Results associated with Vesemnogene Lantuparvovec

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