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Rare Disease Day 2025: Rally for Rare Event Recap

Conversations in Drug Development Trends

On the last day of February every year is Rare Disease Day , a dedicated day to celebrate the rare disease community, including patients, families, caregivers, clinicians, researchers, regulators, and more. For any collaboration inquiries or questions about the 2025 Rally for Rare event, reach out to rare@worldwide.com.

Disease 69
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Scientists create next generation of tools in battle against brain disease

Broad Institute

Scientists create next generation of tools in battle against brain disease By Corie Lok May 21, 2025 Breadcrumb Home Scientists create next generation of tools in battle against brain disease The findings contained in eight studies could lead to targeted gene therapies for brain disorders.

Disease 72
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The Impact of Enzymatic DNA Synthesis on Compact Protein Circuitry for Regulating Protein Secretion Across Multiple Levels of the Central Dogma

Fierce BioTech

The tools developed function entirely post-translationally, enabling synergistic strategies with engineerable regulation across multiple levels of the central dogma. The tools developed function entirely post-translationally, enabling synergistic strategies with engineerable regulation across multiple levels of the central dogma.

DNA 64
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Regulatory Guidance for Oligonucleotide Bioanalysis in Drug Development

Alta Sciences

Regulatory Guidance for Oligonucleotide Bioanalysis in Drug Development pmjackson Wed, 02/19/2025 - 21:30 The unique physicochemical properties of oligonucleotides require the use of specialized bioanalytical approaches, with key considerations including selectivity and specificity, sensitivity, stability, and matrix effects.

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FDA Eliminates Animal Testing: Impact on Biotechs

The Premier Consulting Blog

On April 10, 2025, the US FDA announced that it has a long-term plan to eliminate conventional animal testing in drug development, starting with monoclonal antibodies (mAbs).[ We remain ingrained in the regulations to ensure sponsors can optimize their programs and leverage the benefits that might be available to them via new initiatives.

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Cas9 ancestor engineered into a compact genome editing tool

Broad Institute

Cas9 ancestor engineered into a compact genome editing tool By Tom Ulrich May 14, 2025 Breadcrumb Home Cas9 ancestor engineered into a compact genome editing tool Researchers adapt a compact RNA-guided enzyme from bacteria for a variety of DNA editing tasks in human cells. Ten were capable of editing DNA in human cells.

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Building bridges between Denmark-based and Broad researchers

Broad Institute

Building bridges between Denmark-based and Broad researchers By Corie Lok March 27, 2025 Breadcrumb Home Building bridges between Denmark-based and Broad researchers The Novo Nordisk Foundation Center for Genomic Mechanisms of Disease is forging connections between Danish and Broad scientists. Here are two of their stories.