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The challenges and trends of cell & gene therapies 

Drug Discovery World

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. billion, compared to $19.9 Cancer is king .

Therapies 130
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Could GLP-1 receptor agonists treat addiction and dementia?

Drug Discovery World

Medications containing semaglutide such as Ozempic, licensed for diabetes, and Wegovy, for weight loss, are being studied to see if they can treat a range of different conditions, including addiction and dementia. However, these trials won’t be complete until 2026 at the earliest.

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Analysis Life Sciences Thank You What We Expect the FDA to do in August and September 2024

Agency IQ

Rare Pediatric Disease Priority Review Voucher Program Expiration Threat: The FDA’s popular voucher program for rare pediatric diseases is currently scheduled to expire as of September 30, 2024 unless it is renewed by Congress.

FDA 40
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Scaling up: The need for human cells to give better access to cell therapies

Drug Discovery World

Reece Armstrong speaks to Kathryn Golden , SVP Technical Operations and Manufacturing, bit.bio, about novel technologies, and methods to manufacture human cells for therapies so more patients can access these treatments. Cell therapies have emerged as transformative and potentially curative treatments across a range of disease areas.

Therapies 130
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AstraZeneca to Acquire Alexion, Accelerating the Company’s Strategic and Financial Development

The Pharma Data

Dedicated rare disease unit to be headquartered in Boston. Pascal Soriot , Chief Executive Officer, AstraZeneca , said: “ Alexion has established itself as a leader in complement biology, bringing life-changing benefits to patients with rare diseases. This acquisition allows us to enhance our presence in immunology.

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Models of Life

Codon

The earliest ones relied on simple linear regression and attempted to correlate genetic variations with observable traits or disease risks — such as drug metabolization rates or cancer susceptibility. At best, the corporations with the best models released weak versions to the public under non-commercial licenses.

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Pfizer Investor Day Features Significant Number of Pipeline Advances for COVID-19 Programs and Across Numerous Therapeutic Areas

The Pharma Data

27 key programs highlighted, including assets that could potentially contribute revenue by 2025 and others in the 2026-2028 time frame. Major revenue contributions through 2025 anticipated from Oncology, Vaccines, Rare Disease and Inflammation and Immunology. Rare Disease. BNT162 mRNA-based Vaccine Program.

Vaccine 52