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Start-up to study peptides that modulate gene expression

Drug Discovery World

Other potential cancer indications with ongoing in vitro work include breast and ovarian cancer. HBCI’s CEO, Dr Bomi Framroze said: “HBCI is at the forefront of research in using peptides to modulate gene expression. Our first therapeutic target is resistant castration-resistant prostate cancer (CRPC).

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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.

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Phase Ib study proves efficacy of SFX-01 coated tablet formation

Drug Discovery World

In addition, total blood levels of sulforaphane (SFN) and SFN-metabolites were confirmed in the micromolar range, where efficacy is seen in vitro. It delivers the drug at highly relevant blood levels within the timeframes expected of an enteric coated formulation.” No serious adverse events were observed.

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Biologics – The Next Step in Revolutionary Medication

Roots Analysis

With the increased interest and gradual shift of investment from small molecule drugs to biologics and the establishment of several biologics manufacturing companies / biologics CMOs, more than 250 biologic therapies and vaccines have been developed, globally. Like all drugs, biologics are regulated by the FDA.

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Cell-penetrating peptides as a delivery system for oligonucleotides

Drug Discovery World

Emerging as a new treatment option in rare and orphan disease areas, oligonucleotide therapeutics have matured into a drug class with a broad indication spectrum. Oligonucleotides act on the RNA level through different molecular pathways, but there is a major drawback when using them as drugs: their poor bioavailability and cellular uptake.

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Fibrosis research: Advances and challenges 

Drug Discovery World

Wigerinck has taken four drugs (Prezista, Olysio, Jyseleca and Rekambys) to market. Phase I of the clinical program will dose healthy volunteers with single ascending doses of FIB992 before proceeding to a multiple ascending dose study where the drug is administered for a two-week period.

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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

Usually, only a single copy of the gene is required that too from the mother, however, in cases of AS, either the child does not get any copy, or the child receives two copies – each from mother and father. Thus, restoring the presence of this particular gene can help to restore several of the functions of the brain.