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The future of CNS drug development: signs of real progress

Drug Target Review

2 A raft of emerging therapeutic modalities sits at the centre of this boom, spanning advanced biologics, engineered platforms and next-generation small molecules. Molecular engineering approaches Most CNS disorders involve abnormal gene expression, and molecular engineering is one of the most powerful tools to address this issue.

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Shifting the ADC focus from antibody to payload

Drug Target Review

Most are concentrating on the delivery van and not necessarily on the package,” he adds, highlighting the industry’s tendency to focus on antibody engineering while underinvesting in payload innovation. And in the small molecule space, it’s about overcoming pharmacokinetic factors and formulation issues.”

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How GPCR agonists, including antibodies, are shaping the future of metabolic care

Drug Target Review

For example, the team engineered the MC4R agonists as humanized heavy-chain only antibodies with human Fc domains to improve their overall drug-like properties. A suitable pharmacokinetics profile can be achieved with the addition of half-life extending moieties to the molecule.

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Biogen Advances Investigational SMA Therapy to Registrational Trials After Positive Phase 1 Data

The Pharma Data

Salanersen was engineered as an advanced ASO therapeutic, building on Biogen’s experience with SPINRAZA. Phase 1 Study Overview The Phase 1 clinical program was structured as a single ascending dose study to assess safety, tolerability, and pharmacokinetics of salanersen.

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argenx Advances ARGX-119 for Congenital Myasthenic Syndromes

The Pharma Data

Promising Results from Phase 1b Trial The Phase 1b study was a multicenter, randomized, double-blind, placebo-controlled clinical trial specifically designed to assess the safety, tolerability, pharmacokinetics (PK), immunogenicity, and preliminary efficacy of ARGX-119 in individuals with DOK7-related CMS. Peter Ulrichts, Ph.D.,

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When to Slow Down or Hit the Brakes on a Development Program

The Premier Consulting Blog

Pharmacokinetics and pharmacodynamics :Pharmacokinetics (PK) assess how a drug is absorbed, distributed, metabolized, and excreted by the body. GEN – Genetic Engineering and Biotechnology News. For example, a narrow therapeutic window occurs when the difference between the effective dose and the toxic dose is small.

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Targeting the immunotherapy potential of cytokines IL-12 and IL-18 with new advancements in protein engineering

Drug Target Review

At Alkermes , our interdisciplinary team of protein engineers, immunologists, pharmacologists, and analytical scientists is investigating the biology of several immunomodulatory cytokines including IL-12 and IL-18 to develop novel versions of these molecules with the goal of harnessing their therapeutic potential.