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 Vaccine Vaccination Research 147

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

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

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 52

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. How Can Study Protocols Be More Effective?

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

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

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

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

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 Gene 52

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

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

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