Genetic Disease, Life Science and Regulation

Alyftrek: Vertex Expands Cystic Fibrosis Treatment with Triple-Action Combo

XTalks

JANUARY 6, 2025

The US Food and Drug Administration (FDA) has approved Alyftrek (vanzacaftor/tezacaftor/deutivacaftor), a next-in-class triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator, to treat cystic fibrosis (CF) in patients aged six years and older with at least one F508del mutation or another responsive CFTR mutation.

Gene Therapy

Gene Therapy Protein Genetic Disease Gene

Neurocrine Biosciences’ Crenessity Approved as First New Treatment in Decades for Rare Genetic Disorder CAH

XTalks

DECEMBER 18, 2024

Currently, the standard treatment for CAH involves glucocorticoids, which replace cortisol and help regulate hormone levels. While manageable, the condition requires lifelong treatment and monitoring to prevent complications and ensure healthy development.

Genetics

Genetics Hormones Trials Drugs

New White Paper on Advanced Therapies Sets up Developers for Success

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. One chapter discusses how to form early partnerships with regulators.

Development

Development Gene Therapy Clinical Trials Clinical Trials

Webinars

Bridging Innovation & Patient Care: The Growing Role of AI

MORE WEBINARS

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself.

DNA

DNA Gene Gene Silencing Genome

Sanofi’s Enzyme Replacement Therapy Xenpozyme Wins FDA Approval for Rare Disease ASMD

XTalks

SEPTEMBER 12, 2022

Related: FDA’s CDER Introduces New ARC Program to Accelerate Rare Disease Cures. Sanofi picked up its first Xenpozyme approval for the treatment of Niemann-Pick from Japanese regulators in March followed by an approval from European officials in May.

FDA Approval

FDA Approval Genetics Trials Gene

GW Pharmaceuticals’ Epidiolex Receives Approval for Second Rare Disease Indication

XTalks

AUGUST 6, 2020

TSC is a rare genetic disease that affects approximately 1 in 6,000 people. Defects in hamartin or tuberin lead to a loss of regulation of mTOR, resulting in aberrant differentiation and development, which causes the generation of enlarged cells, as seen in TSC brain lesions.

FDA Approval

FDA Approval Protein Containment Genetic Disease

Verily – Janssen collaboration; Enhancing the response in pancreatic cancer; Avrobio gene therapy eradicates toxic substrate; Gut microbiome responsible for Multiple Sclerosis

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

Gene Therapy Gene Immune Response In-Vivo

After Daybue’s Approval: Acadia Pharmaceuticals’ Next Steps for the Rett Syndrome Community

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

FDA Approval Genetics Trials Drugs

Using CRISPR to Edit the Epigenome Might Be Easier Than We Thought

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. These modifications regulate gene expression without altering the sequence or structure of DNA.

DNA

DNA Gene Gene Silencing Genome

Surrogate Endpoints: How to Choose the Best One for Your Rare Disease Trial

XTalks

SEPTEMBER 17, 2020

In rare disease trials, it’s not always feasible to choose clinically-relevant endpoints to measure the efficacy of a new therapeutic. Patients with this disease have a very high level of LDL-C, which increases their risk of coronary artery disease, including heart attack, stroke and atherosclerosis.

Trials

Trials Gene Hormones Clinical Trials

Sebetralstat Gets NDA Accepted by FDA for Hereditary Angioedema

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

Protein Genetics Trials Allergies

Adzynma Wins FDA Approval as First Treatment for Ultra-Rare Blood Clotting Disorder cTTP

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

FDA Approval Gene Therapy Gene Clinical Trials

4 Life Sciences Trends for 2023

XTalks

DECEMBER 29, 2022

The life sciences and healthcare are among the biggest industries globally, and their significance was particularly highlighted during the past couple of years by the COVID-19 pandemic. Given the hyperfocus on the life sciences thanks to COVID, consumers appear to be more autonomous and vocal about their medical demands and choices.

Life Science

Life Science Gene Therapy Gene Manufacturing

Transitioning Between Academia and Industry: Advice from Leading Scientists That Made the Switch

XTalks

OCTOBER 1, 2020

Sooter has also found success in industry, leading and bringing together the work of different teams of researchers at NeuBase to help drive the company’s goal of developing cures and treatments for genetic diseases, including Huntington’s Disease. Funding in the Life Science Industry vs. Academia.

Scientist

Scientist In-Vitro Research Life Science

10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

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.

Gene Therapy

Gene Therapy Gene Trials Clinical Trials

Tryngolza (Olezarsen) Becomes First FDA-Approved Drug for Familial Chylomicronemia Syndrome

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

FDA Approval Drugs Clinical Trials Clinical Trials

Govorestat Hits Roadblock: FDA Denies Approval for Applied Therapeutics’ Rare Disease Drug

XTalks

NOVEMBER 29, 2024

The lack of existing treatments amplifies the unmet need for a viable therapy. Govorestat is the first therapy evaluated specifically for this ultra-rare condition.

Drugs

Drugs Compliance Clinical Trials Clinical Trials

KalVista Shares New Data on Sebetralstat for Hereditary Angioedema (HAE)

XTalks

FEBRUARY 13, 2025

Kallikrein is a serine protease enzyme that plays a key role in the kallikrein-kinin system by activating kinins, such as bradykinin, which regulate blood vessel dilation and permeability. It is currently under FDA review and, if approved, would be the first oral, on-demand HAE treatment.

Trials

Trials Genetics Allergies Drugs

Clinical Research Informer

Alyftrek: Vertex Expands Cystic Fibrosis Treatment with Triple-Action Combo

Neurocrine Biosciences’ Crenessity Approved as First New Treatment in Decades for Rare Genetic Disorder CAH

Webinars

Trending Sources

New White Paper on Advanced Therapies Sets up Developers for Success

Webinars

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

Sanofi’s Enzyme Replacement Therapy Xenpozyme Wins FDA Approval for Rare Disease ASMD

GW Pharmaceuticals’ Epidiolex Receives Approval for Second Rare Disease Indication

Verily – Janssen collaboration; Enhancing the response in pancreatic cancer; Avrobio gene therapy eradicates toxic substrate; Gut microbiome responsible for Multiple Sclerosis

After Daybue’s Approval: Acadia Pharmaceuticals’ Next Steps for the Rett Syndrome Community

Using CRISPR to Edit the Epigenome Might Be Easier Than We Thought

Surrogate Endpoints: How to Choose the Best One for Your Rare Disease Trial

Sebetralstat Gets NDA Accepted by FDA for Hereditary Angioedema

Adzynma Wins FDA Approval as First Treatment for Ultra-Rare Blood Clotting Disorder cTTP

4 Life Sciences Trends for 2023

Transitioning Between Academia and Industry: Advice from Leading Scientists That Made the Switch

10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

Tryngolza (Olezarsen) Becomes First FDA-Approved Drug for Familial Chylomicronemia Syndrome

Govorestat Hits Roadblock: FDA Denies Approval for Applied Therapeutics’ Rare Disease Drug

KalVista Shares New Data on Sebetralstat for Hereditary Angioedema (HAE)

Stay Connected