article thumbnail

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

Cloudbyz

Biologics: Biologics are large, complex molecules, often proteins, that are produced using living cells. Mode of Action : Small Molecules: They often work by binding to specific sites on target proteins to inhibit or activate their function, which can affect various biological pathways inside the cell.

article thumbnail

Innovative Companies Diving into The Neuro Market Ahead of 2021

The Pharma Data

Vigil’s pipeline strategy is to target these diseases by combining a high-level understanding of microglia physiology, disease genotyping, and patient phenotyping to identify specific genetic variations associated with microglial dysfunction. It’s like taking out the trash.

Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

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

Delveinsight

It also slates to commence a Phase Ib trial of its second candidate, TERN-201, a vascular adhesion protein (VAP-1) inhibitor, with top-line data anticipated in the first half of 2022. Biotheranostics’ two PCR-based genetic tests are well established in the U.S.: Hologic acquires Biotheranostics for USD 230 Million.

RNA 52
article thumbnail

FDA Advisory Committee Recommends Use of Investigational Drug Maribavir (TAK-620) to Treat Post-Transplant Recipients with Cytomegalovirus (CMV) Infection and Disease Refractory to Treatment With or Without Resistance

The Pharma Data

Maribavir, an orally bioavailable anti-CMV compound, is the only antiviral agent presently in Phase 3 development for the treatment of adult post-transplant patients with CMV in SOT or HSCT. Maribavir is the only CMV antiviral drug that targets and inhibits the UL97 protein kinase and its natural substrates. About maribavir.

Drugs 52
article thumbnail

A Complex Chromosomal Abberation Called Adrenoleukodystrophy

Delveinsight

Adrenoleukodystrophy (ALD/ X-ALD) is a rare, X-linked disorder secondary to a mutation in the ABCD1 gene, which encodes for a peroxisomal membrane protein, resulting in an excessive accumulation of very-long-chain fatty acids (VLCFAs) in the brain and adrenal glands. A novel, brain penetrant, orally bioavailable and selective PPAR?

article thumbnail

Revolutionizing Medicine and Public Health: The Emergence of Big Data in Healthcare

Roots Analysis

By analyzing large datasets from various sources, such as clinical trials, genetic data, and EHRs, big data analytics can help identify new potential drug candidates, accelerate the drug discovery process, and lead to the development of more effective and targeted medicines.

article thumbnail

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. What About Small Proteins and Antibodies Reaching Extracellular CNS Targets?

Trials 83