DNA, Gene, Gene Therapy and Genetic Disease - Clinical Research Informer

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Gene Therapy and Pharmacokinetics

Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

Gene Therapy

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Gene therapy specialist bluebird exits “untenable” European market

pharmaphorum

AUGUST 10, 2021

Europe market has proved so hostile to gene therapies when it comes to pricing and reimbursement for gene therapies that bluebird bio has decided to quit the market altogether, according to Andrew Obenshain, president of its severe genetic diseases unit. — Brad Loncar (@bradloncar) August 9, 2021.

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Trending Sources

Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. Both therapies will be available in early 2024.

Gene Therapy

Gene Therapy FDA Approval Gene In-Vivo

Ancient viruses may hold key to gene therapy treatments

Drug Discovery World

DECEMBER 5, 2022

Scientists have unlocked key insights into virus evolution, revealing new information that could help develop treatments for a wide variety of genetic diseases. . Many distinct types of parvoviruses infect mammals – including humans – without causing disease. Parvovirus gene therapy is a cutting-edge biomedical technology.

Gene Therapy

Gene Therapy Gene Genetic Disease Genetics

FDA slaps clinical hold on BioMarin’s PKU gene therapy

pharmaphorum

SEPTEMBER 6, 2021

BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PKU) on hold while it investigates a safety signal.

Gene Therapy

Gene Therapy Gene Genetic Disease DNA

Moderna partners with Life Edit for mRNA gene editing therapies

Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. The company’s nuclease collection includes several Protospacer Adjacent Motifs (PAMs), short sequences that help determine the genome’s DNA segments.

Gene Editing

Gene Editing Gene In-Vivo Gene Therapy

Turning science into business: Amplifying mRNA by targeting regRNAs

Drug Discovery World

APRIL 17, 2024

DDW’s Diana Spencer speaks to Josh Mandel-Brehm , CEO of Massachusetts-based biotech CAMP4, to understand the role regulatory RNAs play in gene expression and how the founders overcame the challenge of launching a business based on brand new science. What are the advantages compared to other gene modulating approaches?

Gene Expression

Gene Expression Gene Genetic Disease RNA

Next Generation Sequencing (NGS) Library Preparation Kits Market: Current Scenario and Future Potential

Roots Analysis

FEBRUARY 21, 2022

Since the introduction of a gene sequencing method by Frederick Sanger in 1977, the field of genomic data collection and analysis has evolved significantly. Advancement in DNA sequencing technologies have resulted in noteworthy developments in various healthcare-related research fields, such as diagnostics and personalized medicine.

Marketing

Marketing Gene Sequencing DNA Genome

Asklepios partners with ReCode on gene-editing platform

Pharmaceutical Technology

JANUARY 10, 2023

Asklepios BioPharmaceutical has entered a research partnership and option agreement with ReCode Therapeutics for exploring its single-vector gene-editing platform. The new solution will enable complete gene insertion by delivering the gene-editing tool and DNA as mixed cargo to desired targets in one LNP.

Gene Editing

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Data demonstrates superiority of circular RNA vs linear mRNA

Drug Discovery World

MAY 15, 2024

In vivo proof-of-concept for Circio Holding’s differentiated circVec platform approach to gene therapy has been demonstrated in two posters at the American Society of Gene & Cell Therapy (ASGCT) 2024 annual meeting. DNA vectors compared to standard linear mRNA-based expression. “We

RNA

RNA In-Vivo Gene Therapy Genetic Disease

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. It’s a great tool for controlling gene expression.”.

DNA

DNA Gene Gene Silencing Genome

Novartis announces lift of partial clinical trial hold and plans to initiate a new, pivotal Phase 3 study of intrathecal OAV-101 in older patients with SMA

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

Clinical Trials Clinical Trials Gene Therapy Trials

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

The future outlook for mRNA therapies

Drug Discovery World

APRIL 30, 2024

Rare diseases are largely treated using gene therapies which employ modified viruses to bring the therapy to the disease cells. However, these therapies can cause severe adverse effects. We believe it can now do the same for rare diseases.” “We

RNA

RNA Protein Vaccination Vaccine

Clinical Catch-Up: December 21-25 | BioSpace

The Pharma Data

DECEMBER 27, 2020

STP705 is a small interfering RNA (siRNA) therapy that leverages a dual-targeted inhibitory property and polypeptide nanoparticle (PNP)-enhanced delivery to knock down both TGF-beta1 and COX-2 gene expression. RESTORE-1 is a Phase II trial of an intracerebral AAV-based gene therapy for Parkinson’s disease.

Trials

Trials Antibody Gene Therapy Clinical Trials

Covid-19 vaccine success bolsters nanoparticle drug delivery research

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. These could feature in the next four to five years.

Drug Delivery

Drug Delivery Vaccination Vaccine Research

Gene Therapy and Pharmacokinetics

Gene therapy specialist bluebird exits “untenable” European market

Trending Sources

Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

Ancient viruses may hold key to gene therapy treatments

FDA slaps clinical hold on BioMarin’s PKU gene therapy

Moderna partners with Life Edit for mRNA gene editing therapies

Turning science into business: Amplifying mRNA by targeting regRNAs

Next Generation Sequencing (NGS) Library Preparation Kits Market: Current Scenario and Future Potential

Asklepios partners with ReCode on gene-editing platform

Data demonstrates superiority of circular RNA vs linear mRNA

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

Novartis announces lift of partial clinical trial hold and plans to initiate a new, pivotal Phase 3 study of intrathecal OAV-101 in older patients with SMA

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

The future outlook for mRNA therapies

Clinical Catch-Up: December 21-25 | BioSpace

Covid-19 vaccine success bolsters nanoparticle drug delivery research

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