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First Patient Dosed in Trial for uniQure’s SOD1-ALS Gene Therapy AMT-162
First Patient Dosed in Trial for uniQure’s SOD1-ALS Gene Therapy AMT-162

October 17th 2024

The phase 1/2 EPISOD1 study, which is taking place in the US, will provide participants with a short immunosuppression regimen before administration of the gene therapy.

Adicet's T-Cell Therapy ADI-100 Snags Clearance for Evaluation in Idiopathic Inflammatory Myopathy and Stiff Person Syndrome
Adicet's T-Cell Therapy ADI-100 Snags Clearance for Evaluation in Idiopathic Inflammatory Myopathy and Stiff Person Syndrome

October 17th 2024

Wave Life Sciences Achieves RNA Editing in Trial for WVE-006 in Alpha-1 Antitrypsin Deficiency
Wave Life Sciences Achieves RNA Editing in Trial for WVE-006 in Alpha-1 Antitrypsin Deficiency

October 17th 2024

Around the Helix: Cell and Gene Therapy Company Updates – October 16, 2024
Around the Helix: Cell and Gene Therapy Company Updates – October 16, 2024

October 16th 2024

Beacon Therapeutics’ XLRP Gene Therapy AGTC-501 Continues to Show Promise in Long-Term Data From SKYLINE Trial
Beacon Therapeutics’ XLRP Gene Therapy AGTC-501 Continues to Show Promise in Long-Term Data From SKYLINE Trial

October 16th 2024

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Beacon Therapeutics’ XLRP Gene Therapy AGTC-501 Continues to Show Promise in Long-Term Data From SKYLINE Trial
Beacon Therapeutics’ XLRP Gene Therapy AGTC-501 Continues to Show Promise in Long-Term Data From SKYLINE Trial

October 16th 2024

Paul Melmeyer, MPP, the executive vice president of public policy & advocacy at MDA
Gene Therapy Will Be a Major Area of Interest for Attendees at MDA’s 2025 Conference

October 10th 2024

Arun Upadhyay, PhD, the chief scientific officer and head of research, development, and Medical at Ocugen
IRD Gene Therapy OCU400’s Phase 1/2 Trial

October 9th 2024

Arun Upadhyay, PhD, the chief scientific officer and head of research, development, and Medical at Ocugen
The Potential of Gene Therapy in Inherited Retinal Disease

October 7th 2024

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