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Jupiter Neurosciences Makes Way in CNS Treatments with $11M IPO

XTalks

Jotrol overcomes these barriers using patented micellar technology, enhancing bioavailability and enabling therapeutically effective doses. CAMP4’s RNA-based therapies focus on genetic diseases like urea cycle disorders, while Rapport’s small molecules aim to address epilepsy, pain and bipolar disorder.

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The New Frontier in Drug Development: Understanding Biologics and Small Molecules

Cloudbyz

Oral Bioavailability: Developing small molecules that are effectively absorbed when administered orally, while also being stable in the digestive system, can be complex. Enhancing bioavailability is often a key focus in the development of small molecule drugs.

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Patient-Centered Innovation for Rare Seizure Disorders: Insights from Marinus Pharmaceuticals

XTalks

CDKL5 deficiency disorder (CDD) is a rare genetic condition caused by mutations in the CDKL5 gene, which is essential for brain development and function. They are exploring a prodrug formulation of ganaxolone to potentially enhance the patient experience and treatment effectiveness by aiming to improve the drug’s bioavailability.

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Ikena bags $120M; Terns raises $87M for NASH; Ribometrix Genentech Collaboration; Hologic acquires Biotheranostics

Delveinsight

The prospects of small molecule binders of RNA over current protein and RNA-based approaches cover potency, selectivity, oral bioavailability, tissue distribution, and central nervous system penetration. Biotheranostics’ two PCR-based genetic tests are well established in the U.S.: Medical technology company Hologic Inc.

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A Complex Chromosomal Abberation Called Adrenoleukodystrophy

Delveinsight

However, the clinical presentation of the disease vary, and neither genetic mutation nor studying the biochemical activity of the brain is sufficient to speculate the phenotypic variation in individuals. A novel, brain penetrant, orally bioavailable and selective PPAR?

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Considerations for Neuroscience Trials with Intrathecal Delivery and Other Methods of Direct CNS Administration

XTalks

A major obstacle for CNS drug developers is the inability to target therapies to the CNS broadly (for instance, a therapy that is orally bioavailable as a pill and has difficulty crossing the blood-brain barrier) and certain CNS regions more specifically. Reporter genes to understand dose and distribution. The patients are waiting!

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Breaking the Waves: How Blue Ocean Strategy in Healthcare Can Propel Pharma / Biotech Companies to Success

Roots Analysis

Blue Ocean Strategy Example: Foundation Medicine has developed a genomic profiling test that helps oncologists identify the best treatment options for their patients based on their genetic makeup. New Drug Formulations as a Blue Ocean Strategy New drug formulations can help improve patient adherence and reduce side effects.