Pharmaceutical Technology

MAY 17, 2023

The deal will see the integration of Scribe’s new CRISPR by Design approach and Prevail’s expertise in developing genetic medicines for neurological disorders for specific genetic targets. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Genetics 130

Genetics Medicine In-Vivo Gene Therapy 130

pharmaphorum

JANUARY 3, 2023

As December 2022 closed out, Pfizer announced positive top-line results from its phase 3 BENEGENE-2 study evaluating fidanacogene elaparvovec (SPK-9001), its investigational gene therapy for treatment of adult males with moderately severe to severe haemophilia B.

Gene Therapy 98

Gene Therapy Gene Medicine Trials 98

STAT News

JULY 11, 2022

Twenty-three years ago, the field of gene therapy was bursting with the promise of breakthrough treatments. Then it was almost instantly derailed by the death of an 18-year-old clinical trial volunteer named Jesse Gelsinger after he received a genetically engineered virus that had been developed to treat his rare liver condition.

Gene 111

Gene Gene Therapy Genetic Engineering Engineer 111

Pharmaceutical Technology

FEBRUARY 13, 2023

Several biotech companies and researchers are now exploring medical devices and gene therapies to address not just common forms of epilepsy, but also rare conditions such as Dravet Syndrome. A genetic treatment for epilepsy Most treatments for epilepsy aim to treat seizures, which is the main symptom of this condition, says Ferraro.

Research 264

Research Gene Gene Therapy Genomics 264

STAT News

AUGUST 18, 2022

Food and Drug Administration approved a new treatment that delivers a potentially permanent, genetic fix for patients with the inherited blood disorder beta thalassemia — and quite possibly a financial lifeline for its manufacturer, Bluebird Bio , STAT tells us.

Gene Therapy 98

Gene Therapy Gene FDA Approval Pharmacy 98

Pharmaceutical Technology

JUNE 4, 2023

Innovation S-curve for the pharmaceutical industry Transcription factors for AAV is a key innovation area in pharmaceutical Adeno-associated virus (AAV) vectors are widely used for gene therapy. There are two main genes in the AAV genome, rep and cap, which encode nine different proteins.

Gene 262

Gene Gene Therapy Genetics DNA 262

Pharmaceutical Technology

JUNE 29, 2022

For cell and gene therapy applications, you need a variety of speciality enzymes of the highest purity, specificity, and consistency. Novozymes has a long legacy of enzyme discovery and the ability to genetically engineer these speciality enzymes to be superior. And it’s within the family – Novozymes!”.

Drugs 295

Drugs Gene Therapy Production Manufacturing 295

Advarra

JUNE 12, 2023

Gene therapy research is exciting and full of promise, but because of the risks involved, it’s also highly regulated, requiring an institutional biosafety committee (IBC) to provide additional oversight and risk assessment. What Does an IBC Review? How is IBC Membership Composed?

Genetic Engineering 98

Genetic Engineering Gene Therapy Genetics Engineer 98

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. Beyond the liver, ReCode’s SORT LNP genetic medicines technology enables the delivery to target cells and organs. By Cytiva Thematic.

Gene Editing 130

Gene Editing Gene Gene Therapy Genetics 130

Advarra

MARCH 31, 2023

The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. A key part of the IBC’s evaluation is assessing the risks posed by the engineered genetic materials.

Engineer 52

Engineer Genetics Clinical Research Research 52

Advarra

AUGUST 8, 2023

Gene therapy research is booming in the clinical setting. In this blog, we summarize the growth, risks, and regulatory requirements for gene therapy research. Defining the Boom in Gene Therapy Research The gene therapy field is experiencing explosive growth in today’s competitive research environment.

Gene Therapy 52

Gene Therapy Gene Research Genetics 52

BioSpace

MAY 10, 2021

Biogen and Capsigen forged a strategic collaboration to engineer novel adeno-associated virus (AAV) capsids that have the potential to become transformative gene therapies that treat underlying genetic causes of various central nervous system and neuromuscular disorders.

Gene Therapy 52

Gene Therapy Gene Engineer Genetics 52

Velocity Clinical Research

OCTOBER 3, 2024

As Nick Spittal states in this Advarra press release, membership in the Gene Therapy Ready (GTR) site network “allows Velocity to start studies over a month faster and provides a meaningful credential and important validation that increases sponsors’ confidence in our specialized capabilities to conduct complex clinical research safely.”

Gene Therapy 52

Gene Therapy Gene Genetic Engineering Clinical Research 52

pharmaphorum

JANUARY 29, 2021

Since that discovery, a flurry of gene-editing focused biopharma companies have launched – including Intellia Therapeutics, CRISPR Therapeutics, Caribou Biosciences and Mammoth Biosciences – and the first drug therapies based on the technology are now in human testing for diseases like cancer. Macrae explains. “So

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

Advarra

JUNE 13, 2024

Recombinant DNA technologies and genetically modified biological agents are being adapted for a wide scope of therapeutic applications, and their use is becoming increasingly common in clinical trials. How can I protect myself from exposure? What should I do if I’m exposed?

Genetic Engineering 52

Genetic Engineering Clinical Trials Clinical Trials Engineer 52

Pharmaceutical Technology

APRIL 5, 2023

But while companies continue studying allogeneic CAR-T therapies, including for their coveted use in solid tumours, such advancements remain challenging. More broadly however, several advancements are on the horizon for cell and gene therapies in 2023. AZ: Cell and gene therapies often come with a high price.

Gene Therapy 147

Gene Therapy Gene Engineer Production 147

Pharmaceutical Technology

SEPTEMBER 29, 2022

Scribe Therapeutics and Sanofi have signed a strategic partnership to expedite the development of breakthrough clustered regularly interspaced short palindromic repeats (CRISPR)-based cell therapies for cancer. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Development 130

Development In-Vivo Genomics Genome 130

Advarra

JUNE 13, 2023

Research in gene therapies and genetically engineered drugs and vaccines are growing exponentially, and will only continue to become more popular. The accelerating gene therapy market is expected to grow globally by 16.6% between 2020-2027.

Gene Therapy 52

Gene Therapy Gene Research Genetic Engineering 52

Scienmag

MARCH 19, 2021

Treatment involving a single injection has long-lasting effects BOSTON – Researchers have used a genetic engineering strategy to dramatically reduce levels of tau–a key protein that accumulates and becomes tangled in the brain during the development of Alzheimer’s disease–in an animal model of the condition.

Genetic Engineering 66

Genetic Engineering Gene Therapy Gene Engineer 66

Cloudbyz

JULY 31, 2024

This revolution is enabling the growth of innovative biomarker-based precision medicine and cell and gene therapy, transforming both clinical research and post-market care. This allows for the selection of patients who are most likely to respond to a particular therapy, optimizing treatment efficacy and minimizing adverse effects.

Gene Therapy 52

Gene Therapy Gene Medicine In-Vitro 52

Pharmaceutical Technology

AUGUST 17, 2022

Biopharmaceutical contract development and manufacturing organisation (CDMO) AGC Biologics has entered a strategic collaboration with RoosterBio to expedite the manufacturing of cell and exosome therapies. These capabilities comprise cell and exosome genetic engineering for expressing therapeutic targets.

Manufacturing 130

Manufacturing Gene Therapy Genetic Engineering Gene 130

Pharmaceutical Technology

AUGUST 25, 2022

The development and usage of innovative technologies, as well as expertise from the selection of cells and genetic modification to intelligent automation of production and information technology systems, will be part of the centre. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Manufacturing 130

Manufacturing Engineer Gene Therapy Production 130

Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

Gene Therapy 52

Gene Therapy Gene Production Clinical Trials 52

Scienmag

JUNE 9, 2021

Credit: RCSI Scientists have developed polypeptide-based materials that act as effective vectors for delivering gene therapies. The first-of-its-kind platform enables the vectors to be adapted to suit the specific gene therapy cargo.

Gene Therapy 53

Gene Therapy Gene Scientist Medicine 53

Pharmaceutical Technology

FEBRUARY 23, 2023

The collaboration will combine the mRNA platform of Moderna with the gene editing technologies suite, including the base editing capabilities of Life Edit for the development of curative therapies to treat challenging genetic diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Gene Editing 147

Gene Editing Gene In-Vivo Gene Therapy 147

XTalks

FEBRUARY 25, 2025

Mirum Pharmaceuticals, a biotech innovator known for developing therapies for rare metabolic disorders, now has FDA approval for its new treatment, Ctexli (chenodiol) tablets the first and only medication approved for cerebrotendinous xanthomatosis (CTX) in adults.

FDA Approval 59

FDA Approval Gene Therapy Gene Genetics 59

Scienmag

DECEMBER 3, 2020

A new study has found that a novel T cell genetically engineered by University of Arizona Health Sciences researchers is able to target and attack pathogenic T cells that cause Type 1 diabetes, which could lead to new immunotherapy treatments.

Genetic Engineering 40

Genetic Engineering Engineer Genetics Bacteria 40

Scienmag

MAY 11, 2021

Proof-of-principle research shows that genes can be accurately edited in cells throughout the body Credit: Zhiqian Li Researchers at the University of California San Diego have laid the groundwork for a potential new type of gene therapy using novel CRISPR-based techniques.

Gene Therapy 63

Gene Therapy Genetics Gene Research 63

STAT News

NOVEMBER 4, 2022

The clinical trial was to be the first time anyone got a gene editing therapy for muscular dystrophy. It was the first time that a gene editing therapy was custom-made to address a single individual’s unique genetic mutation. Epigenome editing aims to dial the expression of genes up or down.

Gene Editing 105

Gene Editing Gene Gene Therapy Scientist 105

Scienmag

MARCH 1, 2021

The in-person Cell Culture Engineering XVII Conference has been postponed until October 16 – October 22, 2021 in Tucson, Arizona. To highlight the key role that the CCE community plays to free people worldwide from the shackles of the COVID-19 pandemic, we are planning two webinars during the original dates of the conference.

Engineer 43

Engineer Gene Therapy Gene Genetics 43

BioTech 365

AUGUST 11, 2020

FDA grants Orphan Drug Designation to Neurogene’s adeno-associated virus vector with engineered transgene encoding the human CLN7 gene NEW YORK–(BUSINESS WIRE)–Neurogene Inc.,

Gene Therapy 40

Gene Therapy Gene Engineer Drugs 40

Advarra

AUGUST 10, 2023

Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. Peter Marks, head of FDA’s CBER – the organization responsible for regulating gene therapies.

Gene Therapy 52

Gene Therapy Development Gene Drugs 52

Scienmag

JUNE 2, 2021

Researchers create novel CRISPR-based fly species as a new method of controlling gene drive spread Credit: Akbari lab, UC San Diego CRISPR-based technologies offer enormous potential to benefit human health and safety, from disease eradication to fortified food supplies.

Gene 98

Gene Engineer Development Research 98

XTalks

JANUARY 26, 2024

This technology, which allows for precise editing of DNA at specific locations, has been a major focus in the field due to its potential to directly target and modify cancer-causing genes. By editing these genes, researchers can effectively neutralize their cancer-promoting effects.

Research 118

Research Gene Editing Gene Genetics 118

XTalks

SEPTEMBER 27, 2023

Poseida is a clinical-stage biopharmaceutical firm that utilizes its unique non-viral gene engineering methods to develop innovative cell and gene therapies. Scientists working at Poseida Therapeutics.

Life Science 59

Life Science Genetic Engineering Engineer Gene Therapy 59

pharmaphorum

OCTOBER 4, 2022

AstraZeneca’s rare disease firm Alexion is set to expand its genomic medicine portfolio with the acquisition of gene editing specialist LogicBio Therapeutics, in a deal worth approximately $68 million.

Gene Editing 64

Gene Editing Gene Genomics Genome 64

XTalks

JANUARY 2, 2024

For instance, Vyjuvek , the first FDA-approved gene therapy for DEB, is priced at $24,250 per vial. a biotech company specializing in the development and commercialization of genetic medicines for rare diseases, announced FDA approval for Vyjuvek for the treatment of DEB. percent) compared to the control group (16.3

FDA Approval 111

FDA Approval Gene Therapy Gene Trials 111