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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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EHA 2023: Kura Oncology’s menin inhibitor ziftomenib shows impressive Phase I results

Pharmaceutical Technology

Ziftomenib targets the menin-KMT2a protein-protein interaction that may occur in patients with KMT2ar or NPM1m genetic alterations. The aberrant protein complex is essential for regulating leukaemia-promoting gene expression. Thus, targeting menin may be an effective strategy for patients with high unmet needs.

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Alnylam identifies gene to lower abdominal fat and decrease cardiovascular risk

Pharmaceutical Technology

Alnylam has reported that it aims to develop a therapy that targets the INHBE gene and reduces the overall risk of T2D and coronary heart disease for at-risk patients. Targeting gene expression that results in increased abdominal fat and leads to obesity is a key factor for the reduction of T2D and wider metabolic syndrome.

Gene 130
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Angelman Syndrome Market: Insights Into The Recent Late-Stage Drug Failures And Novel Approaches To Treating This Rare Neurogenetic Disorder

Delveinsight

The present Angelman syndrome treatment market revolves around the management of symptoms rather than curing the condition. . Several pharmaceutical and biotech companies in the Angelman syndrome market are exploring the novel curative approaches.

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AZ, Scorpion strike deal on elusive transcription factor targets in cancer

pharmaphorum

AstraZeneca has expanded its efforts to develop new therapies targeting transcription factors – proteins that regulate processes in the cell that have become a hot topic for drug discovery in cancer and other diseases.

Protein 59
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International Women’s Day 2024: A Spotlight on 5 Women’s Health Webinars with Xtalks

XTalks

Industry professionals will explore the market, innovative formulations and nutritional solutions tailored to empower women toward optimal health and well-being. A significant 58 percent of women worldwide have heightened their use of supplements to support overall wellness.

Genetics 115
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Molecular Diagnostic Tests: The Unsung Hero of Precise Clinical Decision Making

Roots Analysis

It is worth mentioning that molecular diagnostics is a subset of the broader in vitro diagnostics market. According to the World Health Organization, there are over 40,000 in vitro diagnostics products available in the market. Such test can be used for gene expression profiling, genotyping and detecting chromosomal abnormalities.