Gene Therapy, Genetic Disease and Protein - Clinical Research Informer

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

Gene Therapy Gene Gene Editing Genetics

Bayer gets go-ahead for Huntington’s Disease gene therapy trial

Drug Discovery World

AUGUST 23, 2022

A Phase I/II clinical trial targeting a treatment for Huntington’s Disease (HD) using a gene therapy has been given the clearance to go ahead in France. . A rare inherited neurodegenerative disease, it’s thought to affect 62,000 people in the European Union.

Gene Therapy

Gene Therapy Gene Trials Clinical Trials

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. How do Casgevy and Lyfgenia Work?

Gene Therapy

Gene Therapy FDA Approval Gene In-Vivo

Vyjuvek Gets FDA Nod as First Topical Gene Therapy for Rare Skin Disease

XTalks

MAY 26, 2023

Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB).

Gene Therapy

Gene Therapy Gene FDA Approval Dermatology

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. The post FDA slaps clinical hold on BioMarin’s PKU gene therapy appeared first on.

Gene Therapy

Gene Therapy Gene Genetic Disease DNA

Sarepta says early filing for DMD gene therapy is back on

pharmaphorum

AUGUST 1, 2022

Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD) in the next few months, with a view to making it available in sometime around the middle of 2023.

Gene Therapy

Gene Therapy Gene Trials Genetics

EMA starts rapid review of Bluebird’s gene therapy for rare disease CALD

pharmaphorum

OCTOBER 4, 2020

Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after the EMA started an accelerated review. CALD is caused by mutations in the ABCD1 gene located on the X chromosome, which provides instructions for the production of the ALD protein.

Gene Therapy

Gene Therapy Gene Genetic Disease Protein

MiNA and BioMarin partner to speed development of RNAa therapies

Pharmaceutical Technology

APRIL 26, 2023

MiNA Therapeutics has entered into a research collaboration and option licensing agreement with BioMarin Pharmaceutical to speed up the development of therapeutic ribonucleic acid activation (RNAa) candidates to treat rare genetic diseases. The new deal excludes oncology and other therapeutic areas outside the scope of genetic disease.

Development

Development Genetic Disease Genetics Gene Therapy

World’s first engineered B cell therapy enters human trials

Drug Discovery World

DECEMBER 19, 2023

Developer Immusoft has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for the therapy, designated ISP-001, in this indication. MPS I is a rare, genetic disease that affects the body’s ability to produce the enzyme alpha-L-iduronidase (IDUA).

Engineer

Engineer Trials Genetic Disease Gene Therapy

Turning science into business: Amplifying mRNA by targeting regRNAs

Drug Discovery World

APRIL 17, 2024

JMB: Regulatory RNAs (or regRNAS) are molecules that directly control the expression of nearby protein-coding genes. Recently, research published by Dr Richard (Rick) Young (Whitehead Institute) describes the key mechanism of how these regRNAs regulate gene expression. What diseases are you currently targeting?

Gene Expression

Gene Expression Gene Genetic Disease RNA

Merck and Orna partner for RNA technology-based vaccines and therapies

Pharmaceutical Technology

AUGUST 17, 2022

oRNA molecules have been demonstrated to possess increased stability in vivo compared to linear mRNA and can potentially create more quantities of therapeutic proteins within the body. . Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

RNA

RNA Vaccination Vaccine In-Vivo

Research reveals therapeutic potential of mRNA in rare diseases

Drug Discovery World

JANUARY 11, 2024

The team, led by University College London (UCL), King’s College London and Moderna scientists, found that mRNA could be used to correct a rare genetic liver disease known as argininosuccinic aciduria in a mouse model. mRNA contains instructions that direct the cells to make proteins.

Research

Research Gene Therapy RNA Gene

Tevard Biosciences and Vertex partner to develop tRNA therapies for DMD

Pharmaceutical Technology

MARCH 1, 2023

“By combining Tevard’s ability to restore the production of critical proteins with Vertex’s clinical, regulatory, and manufacturing expertise, we hope to make an important difference for patients and their families.” The company is pioneering tRNA-based therapeutics for modulating mRNA function and curing several genetic diseases.

Development

Development Gene Therapy Genetics Genetic Disease

Shape Therapeutics-Roche’s Deal; AllStripes Raises $50M; Datavant-Real Chemistry’s Partnership; BlueWillow’s Nasal Vaccine

Delveinsight

AUGUST 26, 2021

Shape Therapeutics inks gene therapy deal with Roche worth up to USD 3 Billion. Seattle biotech firm Shape Therapeutics has signed a deal potentially exceeding USD 3 billion with pharma giant Roche to bolster the development of gene therapies for Alzheimer’s and Parkinson’s disease.

Vaccination

Vaccination Vaccine Gene Therapy RNA

Bayer trumpets $1bn CRISPR deal with Mammoth Bio

pharmaphorum

JANUARY 10, 2022

Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences. CRISPR drugs can be used to modify the expression of disease-associated proteins in the body, for example, by correcting a mutation in a specific gene.

In-Vivo

In-Vivo Gene Editing Gene Therapy Gene

Birinapant licensing; AvantGen, IGM pairs up for anti-SARS-CoV-2 antibodies; BeiGene, Novartis to co-dvelop Tislelizumab; Valo raises USD190 M; Bluebird Bio Spins-off

Delveinsight

JANUARY 12, 2021

Birinapant is a clinical-stage SMAC mimetic that degrades Inhibitors of Apoptosis Proteins (IAPs) by binding to them, ultimately leading to cell death in tumor cells. Bluebird spins off to two companies, cleaving off its gene therapy, and cancer units. and Europe.

Licensing

Licensing Licensing Antibody Gene Therapy

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

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

FDA Approval Gene Therapy Gene Clinical Trials

Biopharma Money on the Move: October 21-27

The Pharma Data

OCTOBER 27, 2020

Former Sarepta Therapeutics executive Bo Cumbo left to launch his new gene therapy company, AavantiBio, with a $107 million Series A. AavantiBio’s strategic partnership with University of Florida’s Powell Gene Therapy Center provide their foundational research in rare genetic disorders. Be Biopharma .

Gene

Gene Gene Therapy Medicine Genetics

Roche to present new data at the ISTH 2021 Congress highlighting long-standing commitment to advancing haemophilia A standard of care

The Pharma Data

JULY 6, 2021

Data will include the final analysis from the phase IIIb STASEY study of Hemlibra® (emicizumab) and updated data from the phase I/II study of SPK-8011, an AAV-based gene therapy in development by Spark Therapeutics (a member of the Roche Group). Roche’s Chief Medical Officer and Head of Global Product Development.

Gene Therapy

Gene Therapy Gene Clinical Trials Clinical Trials

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

BioMarin Announces Positive Phase 3 Gene Therapy Trial Results in Adults with Severe Hemophilia A; Study Met All Primary and Secondary Efficacy Endpoints in One-Year Data Set

The Pharma Data

JANUARY 9, 2021

(NASDAQ: BMRN) today announced positive topline results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. This is the largest global Phase 3 study to date for any gene therapy in any indication, with 134 participants.

Gene Therapy

Gene Therapy Gene Trials Clinical Trials

Meet the industry: Who’s at Drug Discovery 2023?

Drug Discovery World

OCTOBER 12, 2023

Dr Michelle Fraser is Head of Cell & Gene Therapy at global health science solutions company, Revvity. For example, to identify protein structures and even binding pockets from sequence data and modelling potential hits in silico. An insightful way to assess which new areas are gaining traction is to follow the money.

Drugs

Drugs Life Science Genome Genomics

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

XTalks

MAY 4, 2021

The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. Epigenetic Editing with CRISPR.

DNA

DNA Gene Gene Silencing Genome

Clinical Catch-Up: December 21-25 | BioSpace

The Pharma Data

DECEMBER 27, 2020

The drug is a topical ointment applied to the lower lid to address the build-up and shedding of proteins at the opening of the Meibomian gland. TSHA-101 is an investigational gene therapy designed to deliver the HEXA and HEXB genes driven by a single promoter within the same AAV9 construct, dubbed a bicistronic vector.

Trials

Trials Antibody Gene Therapy Clinical Trials

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

XTalks

MAY 4, 2021

The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. Epigenome Editing with CRISPR.

DNA

DNA Gene Gene Silencing Genome

Novartis Provides Update on AVXS-101 Intrathecal Clinical Development Program

The Pharma Data

SEPTEMBER 22, 2020

Novartis Gene Therapies to initiate new pivotal confirmatory study to evaluate use of AVXS-101 intrathecal (I T ) formulation in older patients with SMA to further support registration. Novartis Gene Therapies remains confident in the overall benefit-risk profile for patients on treatment.

Clinical Development

Clinical Development Gene Therapy Development Gene

Advances in neuroscience drug discovery

Drug Discovery World

FEBRUARY 22, 2024

For diseases as serious and debilitating for patients and their families as leukodystrophies, learning about such advances in knowledge carries a great deal of hope, which IRCM warmly welcomes,” adds Dr Jean-François Côté, the IRCM’s President and Scientific Director.

Drugs

Drugs Gene Clinical Trials Clinical Trials

Clinical Research Informer

Gene Therapy and Pharmacokinetics

Bayer gets go-ahead for Huntington’s Disease gene therapy trial

Trending Sources

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

Vyjuvek Gets FDA Nod as First Topical Gene Therapy for Rare Skin Disease

FDA slaps clinical hold on BioMarin’s PKU gene therapy

Sarepta says early filing for DMD gene therapy is back on

EMA starts rapid review of Bluebird’s gene therapy for rare disease CALD

MiNA and BioMarin partner to speed development of RNAa therapies

World’s first engineered B cell therapy enters human trials

Turning science into business: Amplifying mRNA by targeting regRNAs

Merck and Orna partner for RNA technology-based vaccines and therapies

Research reveals therapeutic potential of mRNA in rare diseases

Tevard Biosciences and Vertex partner to develop tRNA therapies for DMD

Shape Therapeutics-Roche’s Deal; AllStripes Raises $50M; Datavant-Real Chemistry’s Partnership; BlueWillow’s Nasal Vaccine

Bayer trumpets $1bn CRISPR deal with Mammoth Bio

Birinapant licensing; AvantGen, IGM pairs up for anti-SARS-CoV-2 antibodies; BeiGene, Novartis to co-dvelop Tislelizumab; Valo raises USD190 M; Bluebird Bio Spins-off

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

Biopharma Money on the Move: October 21-27

Roche to present new data at the ISTH 2021 Congress highlighting long-standing commitment to advancing haemophilia A standard of care

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

BioMarin Announces Positive Phase 3 Gene Therapy Trial Results in Adults with Severe Hemophilia A; Study Met All Primary and Secondary Efficacy Endpoints in One-Year Data Set

Meet the industry: Who’s at Drug Discovery 2023?

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

Clinical Catch-Up: December 21-25 | BioSpace

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

Novartis Provides Update on AVXS-101 Intrathecal Clinical Development Program

Advances in neuroscience drug discovery

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