XTalks

DECEMBER 13, 2023

Bolstered by world-class R&D capabilities, a highly productive scientific platform and an established management team, Alpine is dedicated to crafting first- or best-in-class multifunctional immunotherapies through distinctive protein engineering technologies, all aimed at enhancing the quality of life for patients. million and $38.8

Genetics 111

Genetics Vaccination Vaccine DNA 111

XTalks

MAY 4, 2021

To develop CRISPRoff, the researchers generated a single “dead” Cas9 fusion protein capable of mediating DNA methylation and repressive histone modifications, but devoid of any DNA cleavage function. Epigenetic Editing with CRISPR. It’s a great tool for controlling gene expression.”. pyogenes dCas9.

DNA 98

DNA Gene Gene Silencing Genome 98

The Pharma Data

OCTOBER 14, 2020

Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases.

Protein 52

Protein RNA Production Genetics 52

The Pharma Data

DECEMBER 3, 2020

Stoke believes that STK-001, a proprietary antisense oligonucleotide (ASO), has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome. protein expression by leveraging the non-mutant (wild-type) copy of the SCN1A gene to restore physiological Na V 1.1

Medicine 52

Medicine Genetics Clinical Trials Clinical Trials 52

The Pharma Data

JULY 6, 2021

Haemophilia A is a serious, inherited bleeding disorder in which a person’s blood doesn’t clot properly, as they either lack or do not have enough of a clotting protein called factor VIII. The Food and Drug Administration (FDA) granted orphan-disease designation and breakthrough therapy designation in the U.S.,

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

The Pharma Data

AUGUST 3, 2021

SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement. Source link: [link] /.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

XTalks

MAY 4, 2021

To develop CRISPRoff, the researchers generated a single “dead” Cas9 fusion protein capable of mediating DNA methylation and repressive histone modifications, but devoid of any DNA cleavage function. Epigenome Editing with CRISPR. It’s a great tool for controlling gene expression.”. pyogenes dCas9.

DNA 52

DNA Gene Gene Silencing Genome 52

The Pharma Data

OCTOBER 28, 2020

The interim analysis did not include data from other exploratory outcome measures such as seizure frequency, sleep diaries, EEG patterns, UBE3A protein levels in the CSF, ambulation by wearable device, and adaptive behaviors. This condition is typically not inherited but instead occurs spontaneously. About Ultragenyx.

Protein 40

Protein Production Development Drugs 40

The Pharma Data

JANUARY 9, 2021

The most common adverse events associated with valoctocogene roxaparvovec occurred early and included transient infusion-associated reactions and transient, asymptomatic, and mild to moderate rise in the levels of certain proteins and enzymes measured in liver function tests with no long-lasting clinical sequelae. GENEr8-1 Study Description.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52

The Pharma Data

SEPTEMBER 22, 2020

About Zolgensma ® ( onasemnogene abeparvovec ) Zolgensma ® is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time IV infusion.

Clinical Development 52

Clinical Development Gene Therapy Development Gene 52