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Hematological Malignancies Detected in Some Patients Treated With bluebird bio’s Active Cerebral Adrenoleukodystrophy Gene Therapy Eli-Cel
Hematological Malignancies Detected in Some Patients Treated With bluebird bio’s Active Cerebral Adrenoleukodystrophy Gene Therapy Eli-Cel

October 15th 2024

Seven of 67 patients treated with eli-cel across multiple clinical trials have developed hematologic malignancies, according to findings published in The New England Journal of Medicine.

Benitec Biopharma’s Oculopharyngeal Muscular Dystrophy Gene Therapy Produces Clinical Improvements in Swallowing in Phase 1b/2a Trial
Benitec Biopharma’s Oculopharyngeal Muscular Dystrophy Gene Therapy Produces Clinical Improvements in Swallowing in Phase 1b/2a Trial

October 15th 2024

Taysha Seeks to Overturn Natural History Trends With Rett Syndrome Gene Therapy in Dual Phase 1/2 REVEAL Studies
Taysha Seeks to Overturn Natural History Trends With Rett Syndrome Gene Therapy in Dual Phase 1/2 REVEAL Studies

October 14th 2024

Daniela van Eickels, MD, PhD, MPH, the vice president and head of medical affairs for Bristol Myers Squibb’s Cell Therapy Organization
The Role of Established Pharmaceutical Companies in Bringing Cell Therapy to Autoimmune Disease

October 14th 2024

Nanoscope Announces BLA Submission Plans for Retinitis Pigmentosa Gene Therapy MCO-010 Following Meeting With FDA
Nanoscope Announces BLA Submission Plans for Retinitis Pigmentosa Gene Therapy MCO-010 Following Meeting With FDA

October 13th 2024

Video Interviews

Conference Coverage

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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

Daniel Hart, PhD, the senior director and head of technology development at Epic Bio
CRISPR-Mediated In Vivo Epigenomic Activation

October 3rd 2024

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