Drug Discovery World

JANUARY 11, 2024

Patients affected by the disease are found to also experience an imbalance of glutathione regulation, which is important for liver detoxification. We believe it can now do the same for rare diseases.” We aim to apply this approach to other inherited liver diseases and translate mRNA therapy to patients, especially in children.”

Research 59

Research Gene Therapy RNA Gene 59

XTalks

OCTOBER 7, 2020

A tiny child with a devastating genetic disease who wasn’t supposed to blow out the candles on his first birthday cake. Not only did this baby survive to do all these things, but he became a poster child for gene therapy with the regulators at the U.S. Partnering with regulators early.

Development 98

Development Gene Therapy Clinical Trials Clinical Trials 98

Drug Discovery World

JANUARY 30, 2024

Viral vector manufacturing At the Advanced Therapies Awards ceremony, Dr Luigi Naldini, Director of the San Raffaele Telethon Institute for Gene Therapy, was awarded a Lifetime Achievement Award in recognition of ‘extraordinary contributions to gene therapy’, a field he has helped shape through his pioneering work on lentiviral vectors.

Gene Therapy 71

Gene Therapy Manufacturing Gene Clinical Trials 71

Drug Discovery World

APRIL 17, 2024

Recently, research published by Dr Richard (Rick) Young (Whitehead Institute) describes the key mechanism of how these regRNAs regulate gene expression. CAMP4 is exploiting these regulatory interactions to specifically control the expression of genes tied to disease. What diseases are you currently targeting?

Gene Expression 59

Gene Expression Gene Genetic Disease RNA 59

XTalks

DECEMBER 29, 2022

For example, on the pharma and biotech side, there has been growing demand for new drugs and biologics, from new classes of dual-action diabetes and weight loss drugs like Ozempic and Mounjaro to life-saving gene therapies. 1. Cell and Gene Therapies. Here are four life sciences trends to look out for in 2023.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

pharmaphorum

OCTOBER 25, 2022

Astellas has been having some safety-related issues with its gene therapy programmes of late, but it shows no signs of lessened enthusiasm for the category – as a new alliance with US biotech Taysha demonstrates. ” The post Astellas makes another gene therapy play, takes stake in Taysha appeared first on.

Gene Therapy 104

Gene Therapy Gene Genetic Disease Genetics 104

pharmaphorum

AUGUST 12, 2020

The FDA has started its review of Israeli biotech Protalix BioTherapeutics and partner Chiesi’s Fabry disease therapy pegunigalsidase alfa, setting up a possible approval by 27 January. Market research firm Optima Insights has predicted that sales of Fabry disease drugs will more than double from around $1.8

Drugs 98

Drugs Genetic Disease Gene Therapy Trials 98

The Pharma Data

AUGUST 3, 2021

SVP, Chief Medical Officer, Novartis Gene Therapies. “We Additionally, STEER will add to the clinical data and emerging real-world evidence for the use of gene therapy to treat SMA. This route of administration has the potential to open up access for older patients to all the benefits of gene therapy.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

The Pharma Data

SEPTEMBER 22, 2020

Novartis Gene Therapies to initiate new pivotal confirmatory study to evaluate use of AVXS-101 intrathecal (I T ) formulation in older patients with SMA to further support registration. Novartis Gene Therapies remains confident in the overall benefit-risk profile for patients on treatment.

Clinical Development 52

Clinical Development Gene Therapy Development Gene 52

XTalks

NOVEMBER 10, 2023

cTTP is a very rare, inherited and life-threatening blood clotting disorder caused by a disease-causing mutation in the ADAMTS13 (A disintegrin and metalloproteinase with thrombospondin motifs 13) gene, which encodes the ADAMTS13 enzyme that regulates blood clotting by cleaving the von Willebrand factor (VWF) protease.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

XTalks

MAY 4, 2021

These modifications regulate gene expression without changing the sequence or structure of DNA. The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies.

DNA 98

DNA Gene Gene Silencing Genome 98

The Pharma Data

NOVEMBER 19, 2020

He has pioneered the establishment of diagnostic evaluation algorithms for children with sensorineural hearing loss and developed a next generation sequencing platform to determine the genetic causes of hearing loss in children. He has 92 peer review articles published with the majority related to hearing loss. www.sensorion-pharma.com.

Genetics 52

Genetics Containment Genetic Disease Gene Therapy 52

Drug Discovery World

APRIL 30, 2024

In preclinical mice studies, the teams discovered that mRNA could correct a rare genetic liver disease known as argininosuccinic aciduria, which affects how the body breaks down protein and which can lead to high levels of ammonia in the blood. We believe it can now do the same for rare diseases.” “We

RNA 52

RNA Protein Vaccination Vaccine 52

XTalks

MAY 4, 2021

These modifications regulate gene expression without altering the sequence or structure of DNA. The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies.

DNA 52

DNA Gene Gene Silencing Genome 52

Drug Discovery World

FEBRUARY 22, 2024

For diseases as serious and debilitating for patients and their families as leukodystrophies, learning about such advances in knowledge carries a great deal of hope, which IRCM warmly welcomes,” adds Dr Jean-François Côté, the IRCM’s President and Scientific Director.

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59

pharmaphorum

DECEMBER 6, 2020

The trials are the first to test a CRISPR/Cas9 gene editing therapy in humans for a genetic disease, according to the partners. The success of the study is a shot across the bows of companies trying to develop other genetic therapies for thalassaemia and SCD.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

Pharmaceutical Technology

APRIL 24, 2023

Conde says the use of nanoparticle delivery for gene therapies has become more common over recent years. Nanoparticles can be tailored to target specific cells or tissues, release gene therapies in a regulated manner, reduce toxicity, and increase stability,” he added.

Drug Delivery 147

Drug Delivery Vaccination Vaccine Research 147