Pharmaceutical Technology

JANUARY 17, 2023

OR-449 is a first-in-class, orally bioavailable, potent and selective small molecule antagonist of the orphan nuclear receptor steroidogenic factor-1 (SF-1, NR5A1). In paediatric ACC, the transcription factor is commonly amplified at the gene level. SF-1 is an important transcription factor for adrenal gland growth and development.

Bioavailability 147

Bioavailability Clinical Trials Clinical Trials Clinical Development 147

Pharmaceutical Technology

MAY 2, 2023

Erasca has received the US Food and Drug Administration’s (FDA) fast track designation (FTD) for ERAS-801 to treat adults with glioblastoma (GBM) with epidermal growth factor receptor (EGFR) gene alterations.

Bioavailability 130

Bioavailability Clinical Development Gene Trials 130

Camargo

JULY 8, 2021

The UCLA researchers measured monoamine oxidase A (MAOA) gene expression in tumors from cancer patients and found that high MAOA expression was associated with shorter survival times and turned to a mouse model to investigate these observations with promising results.

Development 130

Development Bioequivalency Gene Expression Drugs 130

XTalks

JUNE 14, 2023

Furthermore, the bioavailability of the active pharmaceutical ingredient (API) into the cornea is up to four times stronger with EyeSol technology. Tivanisiran is a preservative-free eye drop that uses RNAi technology, specifically gene silencing, to target and control the signs and symptoms of dry eye disease.

Gene Silencing 97

Gene Silencing Drug Delivery FDA Approval RNA 97

pharmaphorum

JANUARY 29, 2021

While tanezumab is an antibody and has to be delivered by subcutaneous injection, AK1780 is orally bioavailable. Lilly and Asahi Kaesi’s interest in developing AK1780 for pain stems from animal studies showing that knocking out the gene for P2X7 receptors in animal models seemed to abolish the pain response.

Licensing 52

Licensing Licensing Bioavailability Antibody 52

XTalks

AUGUST 30, 2022

We know that a lot of different pathologies, especially as we’ve entered the era of molecular medicine and gene therapies, cause many of the same symptoms and work through final common pathways. Viral Vectors for Gene Delivery to the CNS. Reporter genes to understand dose and distribution. The patients are waiting!

Trials 83

Trials Drug Delivery Drugs Gene 83

Delveinsight

JULY 30, 2020

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?

Hormones 52

Hormones Marketing Gene Trials 52