Drug Discovery World

DECEMBER 5, 2022

Scientists have unlocked key insights into virus evolution, revealing new information that could help develop treatments for a wide variety of genetic diseases. . Many distinct types of parvoviruses infect mammals – including humans – without causing disease. The study was funded by the Bill and Melinda Gates Foundation. . .

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Pharmaceutical Technology

JULY 29, 2022

In preparation for this, drug manufacturing will begin later this year and will be outsourced to external contract manufacturing organisations (CMOs). The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder. David Bumcrot PhD, CSO, CAMP4 Therapeutics.

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Drug Discovery World

MARCH 21, 2023

Although this technique is broadly used, predicting its efficiency when aimed at certain points in the genome can be challenging, making it hard to reliably overexpress certain genes. The post New research begins to write the rules on CRISPR activation appeared first on Drug Discovery World (DDW).

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Drug Discovery World

APRIL 17, 2024

What are the challenges of drug discovery in this area? DS: You say on your website that your approach is applicable to any disease where increasing protein expression is beneficial. What diseases are you currently targeting? JMB: Hundreds of genetic diseases involve a missing or deficient protein and/or enzyme.

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Gene Expression Gene Genetic Disease RNA 59

Roots Analysis

FEBRUARY 21, 2022

Since the introduction of a gene sequencing method by Frederick Sanger in 1977, the field of genomic data collection and analysis has evolved significantly. Moreover, the manual protocols require extensive manipulation, costly reagents and long duration of skilled genomic library production. Advantages of Next Generation Sequencing.

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The Pharma Data

DECEMBER 13, 2020

which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., Dr. Zhang has around 20 years of experience in research and drug development in both industry and academia in the US. 14, 2020 10:00 UTC.

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pharmaphorum

OCTOBER 5, 2022

The new drug candidate for FSHD will combine an RNA molecule from miRecule targeting double homeobox 4 (DUX4) – a protein that is mutated in FSHD – with a nanobody developed by Sanofi that targets muscle cells. .” billion deal in 2018.

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Pharmaceutical Technology

MAY 8, 2023

Innovation S-curve for the pharmaceutical industry Zinc-finger nucleases is a key innovation area in pharmaceutical Zinc finger nucleases (ZFNs) are tools used in genetic engineering to cleave a chosen genomic sequence. ZFNs are also used to create a new generation of genetic disease models called isogenic human disease models.

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Drug Discovery World

FEBRUARY 27, 2023

The rare genetic metabolism disorder called Sanfilippo syndrome Type A- or Mucopolysaccharidosis Type IIIA (MPS-IIIA)- is a genetic disease with devastating effects on the central nervous system affecting around one in 70,000 children.

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Gene Therapy Gene Genetics Genetic Disease 52

XTalks

APRIL 15, 2021

New advances in heart failure genomics are helping to address this challenge. Experts from Servier and Genuity Science recently spoke on a webinar about using genomics data to drive drug development in heart failure and identify new targets for novel therapeutics. Watch this on-demand webinar to hear from these experts.

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Drug Discovery World

MAY 11, 2023

Samsara is leveraging its knowledge of new autophagy biology to rapidly advance multiple drug candidates to the clinic – promising new therapies designed to treat serious rare genetic diseases and neurodegenerative disorders with a strong genetic component.

Life Science 52

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The Pharma Data

JANUARY 17, 2021

n-Lorem Foundation has taken on a challenge that many nations consider too great: treating patients with ultra-rare diseases (which affect 30 or fewer people) for free, for life. That’s not the case with RNA-targeted drugs. They’re basically the same,” but each one uses a particular nucleotide to bind to specific genetic sequence.

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XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

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Drug Discovery World

MARCH 24, 2023

Israeli researchers have developed a drug that is effective in treating a rare genetic muscle disease. The team demonstrated that the disease is caused by a mutation in the gene encoding HMG CoA reductase, inhibiting the enzyme’s activity.

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XTalks

FEBRUARY 14, 2022

The UK’s National Health Service (NHS) has recommended the use of Libmeldy for the treatment of the rare genetic disease metachromatic leukodystrophy (MLD). After further discussion with Orchard Therapeutics, the NHS negotiated an additional, undisclosed discount on the drug, allowing the health agency to now offer it to patients.

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Drug Discovery World

OCTOBER 12, 2023

ELRIG’s Drug Discovery 2023 will be taking place at ACC Liverpool, UK from 18-19 October 2023, focusing on the importance of a diverse life science ecosystem. Such approaches may include cellular technologies for drug discovery, such as a 3D screening model, a topic which will be discussed at Drug Discovery 2023.

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Drug Discovery World

MARCH 6, 2024

To find new ways of diagnosing and treating complex diseases we first must understand the mechanisms underpinning their key pathological drivers, how these relate to different patient subgroups, and which drugs might be useful in ameliorating their effects – this is the basis of precision neuroscience. This is wholly unsurprising.

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XTalks

DECEMBER 13, 2023

The company’s clinical diagnostic division provides a wide array of services, including molecular diagnostic testing, comprehensive genetic testing and high-quality anatomic pathology laboratory services. This substantial investment is accelerating the growth of their new drug pipeline. million and $38.8 million and $33.9

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Drug Discovery World

APRIL 30, 2024

Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. If the body fails to make the right protein, diseases such as cancer, type II diabetes, neurological conditions, and metabolic diseases can occur.

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The Pharma Data

OCTOBER 27, 2020

Food and Drug Administration (FDA). ” The single-reaction panel targets five regions of the viral genome and provides excellent accuracy and sensitivity. “Most real time qPCR-based assays target only two or three targets on the viral genome.” SAN DIEGO , Oct. Darryl Irwin , Vice President of Scientific Affairs.

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Drug Discovery World

FEBRUARY 28, 2023

Finally, dosing and timing may be controlled simply by adjusting the amount of mRNA delivered to the cells, rather than relying on inducible systems that are integrated into the genome. Rare diseases often have few, if any, therapeutic options for their condition.

Genetics 52

Genetics Genetic Analysis Vaccination Vaccine 52

pharmaphorum

APRIL 20, 2022

Cancer is a genetic disease, caused by certain changes in the way that genes control cell function, such as how they grow and divide. Beyond these potential indications, one area that represents a large amount of research and potential is for CRISPR technology to provide new solutions in oncology. Why cancer? The final hurdles.

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The Pharma Data

OCTOBER 28, 2020

These SAEs were assessed as mild or moderate in severity and have generally improved over a period of a few weeks while disease domain improvements have been sustained for three months. The administration method will be modified to help reduce local drug contact time. Food and Drug Administration (FDA). About Ultragenyx.

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