XTalks

MAY 12, 2023

The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. What’s been shown in mouse models of Rett syndrome which also have the genetic disease, is that trofinetide helps strengthen those connections between the neurons. Daybue, also called trofinetide, is a tripeptide.

FDA Approval 97

FDA Approval Genetics Trials Drugs 97

XTalks

SEPTEMBER 16, 2024

These attacks can be unpredictable, often leading to life-threatening situations when they affect the throat or lungs. HAE is caused by a deficiency or dysfunction of the C1-inhibitor, a protein involved in regulating inflammation. HAE affects approximately one in 50,000 people globally, and there is currently no cure for the disease.

Protein 52

Protein Genetics Trials Allergies 52

XTalks

JANUARY 3, 2025

FCS also severely impacts quality of life, causing chronic fatigue and recurrent stomach pain. Tryngolza works by targeting a protein in the liver, apoC-III, which regulates triglyceride metabolism. People with FCS often have triglyceride levels higher than 880 mg/dL, compared to a healthy target level of below 150 mg/dL.

FDA Approval 59

FDA Approval Drugs Clinical Trials Clinical Trials 59

XTalks

SEPTEMBER 25, 2024

Miplyffa primarily slows disease progression using a different mechanism of action, targeting heat shock protein responses to assist cells under stress.

FDA Approval 52

FDA Approval Drugs Protein Gene 52

XTalks

OCTOBER 8, 2024

Apitegromab is a monoclonal antibody that selectively binds to and inhibits myostatin, a protein that limits muscle growth. Current therapies mainly address motor neuron loss, leaving a need for treatments that directly target muscle weakness.

Trials 52

Trials Antibody Genetics Medicine 52

XTalks

NOVEMBER 10, 2023

The US Food and Drug Administration (FDA) has approved Takeda Pharmaceuticals’ Adzynma, the first recombinant protein product for prophylactic (preventive) or on‑demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood clotting disorder.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

XTalks

NOVEMBER 26, 2024

This progressive disease occurs when misfolded transthyretin (TTR) proteins form amyloid deposits in the heart, leading to cardiac dysfunction. BridgeBio has been highly active in the genetic diseases space.

Genetic Disease 105

Genetic Disease Genetics Protein Clinical Development 105

XTalks

APRIL 1, 2025

Through RNA silencing, it targets the expression of antithrombin, a protein that inhibits blood clotting. Moreover, Qfitlia, an siRNA therapy that blocks target protein expression at the RNA level, requires dosing only once every other month, compared to Alhemos daily and Hympavzis weekly dosing.

FDA Approval 59

FDA Approval RNA Clinical Trials Clinical 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

MAY 4, 2021

Related: 2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences. 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.

DNA 98

DNA Gene Gene Silencing Genomics 98

XTalks

DECEMBER 13, 2023

The disease occurs due to a point mutation in the hemoglobin beta globin ( HBB ) gene that codes for one of the proteins that make up hemoglobin, the oxygen carrier in red blood cells. It also affects Hispanic Americans, but at a lower prevalence. The mutation causes red blood cells to develop a crescent or “sickle” shape.

Gene Therapy 98

Gene Therapy Gene FDA Approval In-Vivo 98

XTalks

MAY 4, 2021

Related: 2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences. 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.

DNA 52

DNA Gene Gene Silencing Genomics 52

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.

Genetics 111

Genetics Vaccination Vaccine DNA 111

XTalks

FEBRUARY 13, 2025

HAE is a rare genetic disorder characterized by recurrent episodes of severe swelling (angioedema) in various parts of the body, including the skin, gastrointestinal tract and airways. Without proper treatment, HAE attacks can be painful, debilitating and potentially life-threatening, especially if they involve the airway.

Trials 82

Trials Genetics Allergies Drugs 82

XTalks

JANUARY 6, 2025

Moreover, AI has the potential to identify features and signs of disease that may be overlooked by human experts, offering a new layer of diagnostic capability. Additionally, CRISPR genome-wide screening holds great potential for identifying key disease-associated genes and uncovering novel therapeutic targets.

Gene Therapy 111

Gene Therapy RNA Gene Editing Gene 111

XTalks

FEBRUARY 13, 2025

Mirdametinib is an oral MEK inhibitor that targets the MEK1 and MEK2 proteins, which are part of the RAS/MAPK pathway implicated in the development of NF1-associated tumors. In 2020, AstraZeneca and Merck won FDA approval for Koselugo (selumetinib) to treat NF1-PNs, but its approval was limited to pediatric patients.

FDA Approval 59

FDA Approval Genetics Clinical Trials Clinical Trials 59