BioTech 365

OCTOBER 5, 2020

Diamond Pharma Services has provided EU regulatory, pharmacovigilance, quality and compliance support to GenSight, leading to the Marketing Authorisation Application for LUMEVOQ LUMEVOQ is a gene therapy to treat vision loss due to the rare, mitochondrial genetic disease, Leber Hereditary … Continue reading →

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Gene Therapy Gene Genetic Disease Marketing 52

Drug Discovery World

JANUARY 30, 2024

Dr Naldini open new doors for cures for otherwise untreatable genetic diseases with the full impact of his work still untapped”, she said. Lentivirus strategies are one of the most widely used tools in biomedical research and clinical testing, says Stella Vnook, CEO, Likarda. “Dr

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Gene Therapy Manufacturing Gene Clinical Trials 71

pharmaphorum

OCTOBER 16, 2020

Findacure is a charity that works directly with rare disease patient groups to help them grow and professionalise. Over the last five years we have focused on the power of drug repurposing for rare genetic diseases. This percentage rose to 65% amongst QA, compliance, legal and regulatory professionals.

Clinical Trials 98

Clinical Trials Clinical Trials Trials Life Science 98

Drug Discovery World

JULY 11, 2022

Managing these sensitive materials—including storage, transportation, and tracking—requires robust and adaptable systems to ensure safety, integrity, and regulatory compliance that are required for agency review. There are over 7,000 genetic diseases that could potentially be cured using gene therapy 10. Conclusion.

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Gene Therapy Gene Development Production 52