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CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Pharmaceutical Technology

The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder. Drugs for dialysis, amino acid supplements, and drugs which convert blood ammonia are marketed right now as the go-to urea cycle disorder treatments.

RNA 245
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C4XD and AstraZeneca enter $402m deal to develop respiratory disease therapy

Pharmaceutical Technology

Under the agreement, AstraZeneca will be responsible for the development and marketing of oral therapy for inflammatory and respiratory diseases with a key focus on chronic obstructive pulmonary disease (COPD). A key natural regulator, NRF2 controls antioxidant genesexpression.

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Alnylam identifies gene to lower abdominal fat and decrease cardiovascular risk

Pharmaceutical Technology

Alnylam Pharmaceuticals and collaborators have identified rare mutations in the INHBE gene that is expressed in the liver, related to a lower waist-to-hip ratio for body mass index (BMI), which is often used as an indicator of abdominal fat and is correlated with the risk of type 2 diabetes (T2D) and coronary heart disease.

Gene 130
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US FDA clears Transcenta’s IND for Rett syndrome treatment

Pharmaceutical Technology

The investigational adeno-associated virus (AAV) gene therapy candidate NGN-401 is claimed to be the first to deliver the full-length human MECP2 gene using the company’s Expression Attenuation via Construct Tuning (EXACT) gene regulation technology.

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Global Gene Expression Market Size, Share & Trends Analysis 2021-2028 by Product (DNA Chip/Microarray, Kits & Reagents), Technique (Promoter Analysis, RNA Exp.) – ResearchAndMarkets.com

BioTech 365

Global Gene Expression Market Size, Share & Trends Analysis 2021-2028 by Product (DNA Chip/Microarray, Kits & Reagents), Technique (Promoter Analysis, RNA Exp.) – ResearchAndMarkets.com Global Gene Expression Market Size, Share & Trends Analysis 2021-2028 by Product (DNA Chip/Microarray, Kits & … Continue reading → (..)

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Angelman syndrome Pipeline: Unmet needs call for a robust pipeline

Delveinsight

The necessity of a robust Angelman syndrome pipeline is indispensable at the current moment because of a shortage of any approved therapy option available in the market. Companies like Roche , Biogen , Ovid therapeutics , and Ionis , are among several companies that are finding potential cure taking forward Angelman syndrome market.

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Purple Day 2025: Innovation for Epilepsy

XTalks

It works by increasing SCN1A gene expression, which is crucial for stabilizing brain signaling. Neurostimulation Market Expansion The global neurostimulation device market is projected to reach $22.3 In its Phase I/II trial, patients saw big reductions in seizure frequency.