Delveinsight

FEBRUARY 11, 2021

Verily, Google’s life-science-focused sibling company and Janssen will also seek to tap into the data generated by people during their everyday lives to seek for any previous health-related signals in the two years leading up to the point they consented to participate in the study as well as in the two years after.

Gene Therapy 52

Gene Therapy Gene Immune Response In-Vivo 52

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. The added challenges of advanced therapies in rare-disease clinical trials.

Development 98

Development Gene Therapy Clinical Trials Clinical Trials 98

XTalks

NOVEMBER 27, 2023

Pompe disease, also referred to as acid-maltase disease and glycogen storage disease II, is an uncommon genetic disorder characterized by the gradual weakening of both cardiac and skeletal muscles. The body relies on this enzyme to break down glycogen, a stored form of sugar utilized for energy production.

FDA Approval 104

FDA Approval Clinical Trials Clinical Trials Trials 104

XTalks

SEPTEMBER 22, 2022

Clinical Trial Behind Spevigo. The efficacy of Spevigo (spesolimab) was determined based on the results of the Effisayil 1 double-blind, randomized, Phase II clinical trial. GPP occurs in individuals with specific gene variations as it is a genetic disease , and variations in the genes IL36RN and AP1S3 are known to cause it.

FDA Approval 52

FDA Approval Clinical Trials Clinical Trials Dermatology 52

pharmaphorum

JANUARY 27, 2023

Developing medicines – for people living with disease Dr Mullen’s current role involves providing medical monitoring support, as well as safety, pharmacovigilance, scientific, and medical advice across a range of client projects, including advanced therapies and orphan drugs. I still feel that it is very important,” Dr Mullen said. “We’ve

Clinical Trials 111

Clinical Trials Clinical Trials Trials Development 111

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 12, 2023

Caused by mutations in the MECP2 gene located on the X chromosome, Rett syndrome is primarily an X-linked disorder; however, there have been rare cases of boys being affected, usually with more severe symptoms due to the absence of a second X chromosome. Dr. Bishop: Rett syndrome is a genetic disorder caused by a mutation in the MECP2 gene.

FDA Approval 97

FDA Approval Genetics Trials Drugs 97

XTalks

FEBRUARY 27, 2023

Alpha-mannosidosis is an extremely rare genetic metabolic disease affecting approximately one in 500,000 people. The lysosomal storage disorder is caused by mutations in the MAN2B1 gene, which codes for lysosomal alpha-mannosidase, an enzyme that degrades glycoproteins (proteins attached to sugar residues).

FDA Approval 52

FDA Approval Life Science Containment Genetics 52

pharmaphorum

DECEMBER 8, 2021

Acadia Pharma’s trofinetide has hit its efficacy targets in a phase 3 trial involving girls with the devastating neurological disorder Rett syndrome, although safety has dampened enthusiasm a little. Results from a phase 3 trial of blarcamesine are due next year. At 12 weeks, trofinetide-treated patients had a 5.1-point

Marketing 59

Marketing Drugs Life Science Trials 59

XTalks

DECEMBER 22, 2021

If the past year is anything to go by, then 2022 will also be a year marked by continued innovations in the life sciences. The development and widespread adoption of new technologies is key to revolutionizing the way we diagnose, prevent, treat and manage disease. The RNA Revolution: From mRNA Vaccines to RNA Editing.

Life Science 98

Life Science RNA Vaccination Vaccine 98

XTalks

MARCH 21, 2024

The US Food and Drug Administration (FDA) has awarded approval to Orchard Therapeutics for its gene therapy Lenmeldy (atidarsagene autotemcel) for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).

Gene Therapy 111

Gene Therapy Gene Drugs FDA Approval 111

XTalks

SEPTEMBER 12, 2022

ASMD is a rare genetic lysosomal storage disease caused by mutations in the sphingomyelin phophodiesterase-1 ( SMPD1 ) gene that codes for the acid sphingomyelinase (ASM) enzyme. Patients in the trial received Xenpozyme or placebo for 52 weeks. ASM is required for breaking down a complex lipid called sphingomyelin.

FDA Approval 98

FDA Approval Genetics Trials Regulation 98

The Pharma Data

JANUARY 17, 2021

Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases. Of course, this is how biotech investments work.

In-Vivo 52

In-Vivo Marketing Engineer Genetics 52

XTalks

MARCH 3, 2021

BridgeBio is dedicated to developing therapies for genetic diseases with unmet needs. The FDA remains committed to facilitating the development and approval of safe and effective therapies for patients affected by rare diseases — an area of critical need.”. Nulibry Trial Results.

FDA Approval 64

FDA Approval Genotype Clinical Trials Clinical Trials 64

XTalks

AUGUST 6, 2020

TSC is a rare genetic disease that affects approximately 1 in 6,000 people. The disease usually presents within the first year of life, displaying subtle signs that can take years to develop. Due to this, the disease can be misdiagnosed or not even recognized for a while.

FDA Approval 64

FDA Approval Protein Containment Genetic Disease 64

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). Related: Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data. The cells are then injected back into the patient.

Gene Therapy 98

Gene Therapy Drugs Gene Gene Silencing 98

XTalks

APRIL 15, 2021

million people in the US, yet there is no treatment for the most common form of the disease: heart failure with preserved ejection fraction (HFpEF). A lack of efficacy is the greatest reason for clinical trial failures, especially in Phase II and Phase III,” says Dr. Tyl. Heart failure affects about 6.2

Development 98

Development Drugs Genome Genomics 98

Drug Discovery World

MARCH 6, 2024

Guiding neurological drug discovery and development to success The 20 years since the Human Genome Project has seen transformational advances in the molecular understanding of cancers and rare genetic diseases, leading to genetically informed, personalised selection of therapies and massively improved outcomes for many patients.

Genome 65

Genome Genomics Clinical Trials Clinical Trials 65

XTalks

DECEMBER 13, 2023

The company’s sustained investment in R&D has led to a rapid expansion of its pipeline in 2023, with notable milestones such as four infectious disease vaccines in Phase III trials, including the recently submitted respiratory syncytial virus (RSV) vaccine for regulatory approval.

Genetics 111

Genetics Vaccination Vaccine DNA 111

XTalks

AUGUST 29, 2022

Now a common gene editing tool, the popularity of the CRISPR-Cas9 system has increased over the past decade. CRISPR is notable for engineering living cells, allowing scientists to edit, turn off, delete, or replace genes in a cell’s genome. Marson’s group initially collaborated with CRISPR pioneer Jennifer Doudna, PhD, in 2015.

Gene Editing 98

Gene Editing DNA Gene Genome 98

Drug Discovery World

APRIL 30, 2024

At the start of 2024, the first patients in a clinical trial investigating an mRNA cancer therapy were dosed. The Mobilize trial is investigating the mRNA-4359 therapy and its potential for treating melanoma, lung cancer and other solid tumour cancers. We believe it can now do the same for rare diseases.” “We

RNA 52

RNA Protein Vaccination Vaccine 52

The Pharma Data

OCTOBER 15, 2020

Axovant – Kristin Vuori was named to the board of directors at Axovant Gene Therapies Ltd. Sabbagh will be responsible for expanding Inozyme’s proprietary pipeline by identifying and developing new therapeutics for monogenic and non-genetic diseases of abnormal mineralization. Additionally, Parag V.

In-Vivo 52

In-Vivo Business Development Manufacturing Development 52