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Vertex presents new data on Casgevy in children

Published

2 days ago

June 16, 2026

Sickle cells

Vertex Pharmaceuticals has announced data demonstrating the clinical benefits of Casgevy (exagamglogene autotemcel) in people ages five years and older living with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT).

The results, from pivotal studies in children ages 5–11, show that the efficacy and safety outcomes in this age group are consistent with those established in adult and adolescent patients.

The data were presented at the European Hematology Association (EHA) Congress and simultaneously published in the New England Journal of Medicine (NEJM).

“The data presented at EHA and published in NEJM underscore the consistent, durable and transformative benefits Casgevy can provide to people living with sickle cell disease or transfusion-dependent beta thalassemia from early in life,” said Dr Carmen Bozic, Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex.

Franco Locatelli, Professor of Pediatrics at the Catholic University of the Sacred Heart of Rome, and Director of the Department of Pediatric Hematology and Oncology at Bambino Gesù Children’s Hospital, added: “These data represent a profoundly important step forward, and I look forward to the possibility of providing earlier intervention to prevent complications in children and for families who have had limited potentially curative options to date.”

Casgevy is currently approved for eligible people 12 years and older with SCD with recurrent VOCs or TDT in several countries around the world. In the US the regulatory review is underway with the FDA to expand the use of Casgevy to younger children after Vertex was awarded the Commissioner’s National Priority Voucher.

Vertex has also recently completed regulatory submissions in the Kingdom of Saudi Arabia and UK to expand the use of Casgevy to younger children.

The post Vertex presents new data on Casgevy in children appeared first on Drug Discovery World (DDW).

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Ebola cases increase almost 40% in a week as death toll passes 200

DAKAR, Senegal — The Ebola outbreak in Congo and Uganda has claimed more than 200 lives in its first month and is the worst known outbreak at this stage, with up to 35,000 suspected potential contacts, Africa’s Centres for Disease Control and Prevention said on Thursday.

With 894 confirmed cases so far, the current outbreak is three times worse than a previous outbreak in Uganda in 2000, which had 281 cases at the same point, said Dr. Wessam Mankoula, a medical epidemiologist at Africa CDC.

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Published

40 minutes ago

June 18, 2026

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IDBS and Alchemi Agree to Accelerate AI-Driven Biopharma Regulatory Filings

Published

52 minutes ago

June 18, 2026

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IDBS, which provides cloud software for biopharma, and Alchemi, an applied AI company, partnered to connect AI agents to data captured and contextualized in the IDBS Polar platform across the drug development lifecycle. By capturing such data at the point of creation within a single governed backbone, Polar delivers the data foundation that makes agentic AI viable in regulated environments, explains IDBS in a statement.

Preparing regulatory filings remains one of biopharma’s most persistent bottlenecks. CMC teams spend months assembling data, drafting reports, and reconstructing process histories, while current AI tools often break the compliance chain by pulling data out of validated systems, according to an IDBS spokesperson, adding that connecting Alchemi’s purpose-built agents to Polar’s AI-ready data foundation keeps validated data traceable and auditable.

CMC technical reports, clinical study reports, and submission dossiers can be drafted faster and routed through human-in-the-loop workflows for review and sign-off, maintaining compliance, points out the IDBS official. According to Alchemi, in customer deployments across biopharma, teams have produced documents of this type up to 70% faster using Alchemi’s agents.

“A regulatory filing that takes a team weeks, our agents draft in minutes, at submission-ready quality, with the compliance trail intact, because they work straight from governed data in Polar,” reports Anuj Chadha, co-founder, Alchemi.

“Our mission is to accelerate life-changing therapies to patients, and as part of Danaher, that means turning ideas into impact with speed and certainty,” says Pietro Forgione, general manager, IDBS. “[Our company] delivers a governed data backbone that makes AI-ready data available across the biopharma lifecycle. By partnering with Alchemi, we can accelerate critical regulatory milestones using high-quality, compliant AI agents,” said Pietro Forgione, general manager, IDBS.

The post IDBS and Alchemi Agree to Accelerate AI-Driven Biopharma Regulatory Filings appeared first on GEN – Genetic Engineering and Biotechnology News.