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Chinook and Ionis partner to develop ASO therapy for kidney disease

Pharmaceutical Technology

Antisense therapies are designed for destroying mRNA, reducing the amount of disease-causing protein. They will also have the potential to treat diseases that are caused by too little protein by increasing production, thus restoring the protein to normal levels.

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Italfarmaco’s Duvyzat Wins FDA Approval as First Nonsteroidal Treatment for All Genetic Variants of DMD

XTalks

Duvyzat, a histone deacetylase (HDAC) inhibitor that works to reduce inflammation and muscle loss, is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. Progressive muscle weakness in the disease is caused by genetic mutations in the dystrophin gene that lead to a lack of functional dystrophin protein.

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Frontotemporal dementia: the state of treatment development

Pharmaceutical Technology

Different approaches that are studied include antisense oligonucleotides (ASOs), and gene therapies, which are in early clinical trials. Other lines of research look at the genetic overlap between FTD and amyotrophic lateral sclerosis (ALS), which could be used in the development of treatments for both conditions.

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Plant Host Systems: A Novel Advancement in Protein Expression

Roots Analysis

In the past few years, plants have been gaining a rapid traction, involving genetic modification and expression of new genes in plant host cells. Currently, a large number of plant-based proteins are being evaluated in clinical phases of development, including vaccine candidates against COVID 19 and multiple rare therapeutic disorders.

Protein 52
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FDA grants Fast Track status for Biohaven’s taldefgrobep alfa to treat SMA

Pharmaceutical Technology

Taldefgrobep is a complete human anti-myostatin recombinant protein that is developed for lowering free myostatin. Biohaven Clinical Development vice-president Lindsey Lee Lair said: “We are very pleased the FDA granted Fast Track designation for taldefgrobep alfa for the treatment of SMA.

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Vertex eyes controllable genetic drugs with $1.3bn Obsidian alliance

pharmaphorum

billion in milestone payments in the deal, which will focus on using small-molecule compounds to “fine tune” the effects of genetic medicines in the body. The approach relies on the use of drug responsive domains (DRDs) on the gene-editing medicine, which can be used to either increase or decrease protein expression.

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Gene Therapy and Pharmacokinetics

Camargo

When developing a clinical development program for these increasingly popular therapies, it is important that sponsors use modeling and pharmacokinetic (PK) analysis to evaluate parameters that can be measured while dosing with gene therapy drugs, to characterize exposure-response data and inform rational dosing.