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The company requested priority review for the BLA.

Take a look what stood out as pillars of progress and success from all of CGTLive's most popular lysosomal disorders stories in 2024.

Look back on the 2024 stories that our readers were on top of during the medical meetings covered by the CGTLive team.

Take a look what stood out as pillars of progress and success from all of CGTLive's most popular hematology stories in 2024.

Take a look what stood out as pillars of progress and success from all of CGTLive's most popular cardiology stories in 2024.

Take a look what stood out as pillars of progress and success from all of CGTLive's most popular oncology stories in 2024.

Take a look what stood out as pillars of progress and success from all of CGTLive's most popular neurology stories in 2024.

Take a look what stood out as pillars of progress and success from all of CGTLive's most popular ophthalmology stories in 2024.

Take a look what stood out as pillars of progress and success from all of CGTLive's most popular feature stories in 2024.

In 2024, CGTLIve spoke with experts outside the context of the conference cycle to seek their insight on company announcements, pipeline therapies, and disease awareness, among other topics.

Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado, discussed 5-year follow-up data from the TRANSCEND-NHL-001 clinical trial.

The trial is evaluating AURN001 for the treatment of corneal edema secondary to corneal endothelial dysfunction.

Ben Samelson-Jones, MD, PhD, the associate director of clinical in vivo gene therapy at Children’s Hospital of Philadelphia, discussed follow-up data of up to 6 years with investigations of fidanacogene elaparvovec.

Review top news and interview highlights from the week ending December 20, 2024.

Steven W. Pipe, MD, a professor of pediatric hematology/oncology at CS Mott Children’s Hospital, discussed findings from the open-label extension of the ATLAS studies at ASH 2024.

Natural killer cells could redefine medicine and offer new hope for cancer, autoimmune diseases, and neurological disorders through advanced CAR-NK therapies.

The product will be marketed under the name Ryoncil, and is indicated for patients aged 2 months and older.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The agency agreed that data from the ongoing phase 1/2 studies compared to external control natural history data will be sufficient to support a BLA.

Hosted by Joseph Fraietta, PhD, ImmunoLogic will be tailored for an audience fluent in the language of medicine and biotechnology, offering data-driven insights while maintaining accessibility.

The trial for the exon-51 skipping therapy is already active in the United Kingdom.

Mind Moments®, a podcast from our sister site NeurologyLive®, held an exclusive interview with Jonathan Parker, MD, PhD.

CGTLive took a closer look at the design of the Eli Lilly subsidiary’s trial in PD for the AAV vector-based gene therapy.

Cutting-edge cell and gene therapies have sparked ideas for new payment models that could reshape affordability and access to life-changing care.

Real-world data showed ide-cel was active in patients with central nervous system manifestations of multiple myeloma.

The head of the Referral Center for Sickle Cell Disease and Clinical Research Department at Hôpital Intercommunal de Créteil of the Université Paris Cité discussed the Drepagreffe-1 and 2 studies and improvements seen over 10 years of follow-up.

Review top news and interview highlights from the week ending December 13, 2024.

The associate director of clinical in vivo gene therapy at Children’s Hospital of Philadelphia discussed follow-up data of up to 6 years with investigations of fidanacogene elaparvovec.