Drug Discovery World

NOVEMBER 16, 2023

The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the world’s first gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Casgevy is designed to work by editing the faulty gene in a patient’s bone marrow stem cells so that the body produces functioning haemoglobin. “I

Gene Therapy 111

Gene Therapy Gene Gene Editing Clinical Trials 111

Pharmaceutical Technology

MAY 24, 2023

It can correct several disease elements associated with PKD, a rare, monogenic red blood cell disorder caused by a mutation in the PKLR gene. The RMAT designation programme is intended to accelerate the drug’s development and review processes for products, including gene therapies.

Gene Therapy 130

Gene Therapy Gene Licensing Licensing 130

Pharmaceutical Technology

JUNE 19, 2023

Although only a small number of gene therapies have reached the market thus far, the industry is poised to grow quickly over the next few years. According to GlobalData’s clinical trials database, there are currently 1,231 planned and ongoing trials for gene therapies and gene-modified cell therapies alone.

Gene Therapy 130

Gene Therapy Gene Packaging Packaging 130

Drug Discovery World

DECEMBER 6, 2023

Gene editing company Eligo Bioscience has announced a successful $30 million Series B funding round, led by Sanofi Ventures. “We We are excited to lead this financing for Eligo and support the company as they move to the clinic,” said Laia Crespo, Partner at Sanofi Ventures. “We

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

pharmaphorum

MARCH 10, 2022

18 months after signing its first partnering deal with Genentech, Scenic Biotech has raised $31 million in first-round financing that it will use to take its own genetic modifier-targeted medicines into clinical trials. The post Scenic Bio raises $31m for genetic modifier platform appeared first on.

Genetics 98

Genetics Gene Sequencing Gene Clinical Trials 98

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. . The disease is caused by anomalous repeating mutations in the huntingtin gene leading to abnormal protein aggregates in nerve cells.

Gene Therapy 98

Gene Therapy Gene Trials Clinical Trials 98

XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Eagleton recently spoke on a webinar with his colleagues from Medpace about lessons learned from successful approaches from rare disease and gene therapy product approvals.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

Drug Discovery World

OCTOBER 18, 2023

Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies.

Gene Therapy 64

Gene Therapy Gene Clinical Trials Clinical Trials 64

XTalks

DECEMBER 14, 2023

Advances in precision medicine and immunotherapy offer new hope for targeted treatments. Please tell us more about your role as Senior Director, Clinical Trial Portfolio and Program Management at the Pancreatic Cancer Action Network. Research is ongoing to find better ways to diagnose and treat pancreatic cancer.

Clinical Trials 52

Clinical Trials Clinical Trials Trials Doctors 52

Drug Discovery World

MARCH 8, 2023

If successful, GNT-0003 would be the first gene therapy for Crigler-Najjar syndrome.” PRIME status was granted to GNT-0003 following promising results from early phases of the European trial currently underway in collaboration with the CureCN consortium, and sponsored by Genethon.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

pharmaphorum

JANUARY 29, 2021

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers.

Gene Editing 141

Gene Editing Gene Gene Therapy In-Vivo 141

Drug Discovery World

AUGUST 16, 2023

A new study has shed light on the genetic messages encoded by genes within ‘triple negative’ breast cancers (TNBC), and shows they could predict response to chemotherapy. “Our findings paint a complex picture – with many, dynamic factors driving how tumours respond to treatment.”

Genetics 52

Genetics Gene Hormones RNA 52

XTalks

OCTOBER 26, 2021

After initial rejection from the National Institute for Health and Care Excellence (NICE) last year, the non-departmental public body of the Department of Health in England has now given the green light to the gene silencing treatment Givlaari (givosiran) for the treatment of the rare metabolic disorder, acute intermittent porphyria (AIP).

Gene Silencing 98

Gene Silencing Gene Clinical Trials Clinical Trials 98

Drug Discovery World

DECEMBER 19, 2022

Researchers have found that a topical gene therapy gel helps heal wounds in patients with life-threatening blistering skin disease epidermolysis bullosa (EB). . The results of the trial, which was funded by Krystal Biotech, were published in The New England Journal of Medicine.

Gene Therapy 52

Gene Therapy Gene Dermatology Clinical Trials 52

Roots Analysis

JANUARY 22, 2024

However, these approaches are incompetent for dealing with intrinsic carcinogenic genetic abnormalities. This resulted in the discovery of an individualized concept of treatment known as oncology precision medicine which primarily utilizes the patient’s genomic information to provide gene-targeted diagnostics and therapeutics.

Medicine 40

Medicine Genetics Genome Genomics 40

pharmaphorum

MARCH 24, 2022

There’s a rich history of finding useful medicines from fungi, from the antibiotic penicillin to immune suppressant cyclosporine and cholesterol drug lovastatin. The platform uses an artificial intelligence (AI) algorithm to zero in on the genes encoding natural products based on their computationally predicted human target.

Medicine 89

Medicine Genome Genomics Engineer 89

Camargo

MAY 2, 2021

Advances in scientific knowledge and growth in the cell and gene therapy space have led to a new and exciting era of medicine for patients, as well as a new motivation for regulators to provide clear, efficient pathways for product developers. Background: The Advancement of Cell and Gene Therapies.

Gene Therapy 155

Gene Therapy Gene Packaging Packaging 155

Scienmag

APRIL 28, 2021

Routine genetic screening for all children with cardiomyopathy could make a powerful difference in outcomes and even survival Credit: Douglas Levere/University at Buffalo BUFFALO, N.Y.

Genetics 93

Genetics Gene Clinical Trials Clinical Trials 93

Drug Discovery World

DECEMBER 11, 2023

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is also the first FDA-approved treatment to use CRISPR gene editing technology.

Gene Therapy 52

Gene Therapy FDA Approval Gene Gene Editing 52

The Pharma Data

AUGUST 7, 2020

As the pharma industry stands firm in its commitment to advance the sector to fight Covid-19, news has emerged from the European Commission who intend to streamline the development of therapies using genetically modified organisms to treat Covid-19. Regenerative medicine and advanced therapies thriving despite Covid-19 disruption.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Trials 52

pharmaphorum

OCTOBER 12, 2022

Now, he leads Whitelab Genomics as its artificial intelligence (AI) platform powers the development of genomic therapies – an emerging field in which genetic sequences are injected into cells to target and repair damaged genes. The evolving field of genomic medicine.

Genome 90

Genome Genomics Medicine Development 90

Cloudbyz

MARCH 30, 2023

Cell and gene therapies are rapidly becoming an essential component of modern medicine, offering hope to patients with previously untreatable diseases. However, the development and delivery of cell and gene therapies present significant operational challenges that must be navigated carefully to ensure successful clinical trials.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52

Pharmaceutical Technology

MAY 22, 2023

Vyjuvek is a non-invasive, topical, re-dosable gene therapy that delivers functional human COL7A1 gene copies to offer wound healing. The regulatory approval was supported by data from the GEM-1/2 and GEM-3 clinical trials. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

FDA Approval 130

FDA Approval Gene Therapy Genetic Disease Gene 130

STAT News

AUGUST 12, 2022

… Two children have died from acute liver failure after being administered Zolgensma, a pricey gene therapy sold by Novartis to treat a rare disease , STAT reports. The Well, whatever you do, have a grand time. But be safe. Enjoy, and see you soon. Continue to STAT+ to read the full story…

Gene Therapy 98

Gene Therapy Gene Vaccination Vaccine 98

XTalks

NOVEMBER 7, 2022

Such information can reveal details about tumor staging, tumor progression, heterogeneity, gene mutations and clonal evolution. Liquid Biopsy Use in Oncology Clinical Trials. FoundationOne ® Liquid CDx (Foundation Medicine; end-to-end commercial provider). Bahassi during the webinar and is shown in Table 1.

Clinical Trials 98

Clinical Trials Clinical Trials Trials FDA Approval 98

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

XTalks

JUNE 8, 2021

Novartis’ Zolgensma (onasemnogene abeparvovec) gene therapy has been making significant strides as of late, including dosing of the first Spinal Muscular Atrophy (SMA) patient with the treatment in the UK last week. SMA is caused by mutations in the survival motor neuron 1 ( SMN1) gene, which along with the SMN2 gene, encode the SMN protein.

Gene Therapy 110

Gene Therapy Gene Protein Trials 110

XTalks

FEBRUARY 25, 2022

There has been increasing awareness around the need for improving inclusion and diversity in clinical trials. As such, there has been a growing emphasis on efforts to achieve better and more accurate representations of minority and underrepresented populations like Black people in clinical studies. Race-Based Medicine.

Clinical Trials 52

Clinical Trials Clinical Trials Trials Research 52

Scienmag

MAY 31, 2021

NIH- and USU- led study links ALS to a fat manufacturing gene and maps out a genetic therapy Credit: Courtesy of the NIH/NINDS.

Genetics 56

Genetics Scientist Gene Manufacturing 56

The Pharma Data

JUNE 18, 2023

These results reinforce the impact that innovative medicines can have in the prevention of migraine.” About Lilly Lilly unites caring with discovery to create medicines that make life better for people around the world. There were no new safety findings. Source link: [link]

Clinical Trials 40

Clinical Trials Clinical Trials Trials Medicine 40

Cloudbyz

MAY 19, 2021

Introduction Cell and gene therapy is an upcoming wave of therapeutic innovation in the healthcare and life sciences industry and is being pragmatically accepted worldwide. The gene therapy market reported its first market approvals back in 2017 and the evolution has been extensive ever since.

Gene Therapy 52

Gene Therapy Gene Manufacturing Production 52

Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. billion, compared to $19.9 Cancer is king .

Gene Therapy 52

Gene Therapy Gene Manufacturing Medicine 52

Drug Discovery World

JULY 11, 2022

David Lewandowski, Business Director of Cell & Gene Therapy at Azenta Life Sciences, explains how efficient sample management can help to work efficiently and bring therapies to market faster. The global cell and gene therapy (CGT) market reached a value of ~$4 billion in 2020 and is expected to grow to ~$34 billion by 2030 1.

Gene Therapy 52

Gene Therapy Gene Development Production 52

XTalks

JANUARY 26, 2024

Innovations in Cancer Therapy CRISPR/Cas9, a groundbreaking gene-editing technology, has demonstrated significant potential in oncology, offering new avenues for cancer treatment. By editing these genes, researchers can effectively neutralize their cancer-promoting effects.

Research 118

Research Gene Editing Gene Genetics 118

Scienmag

OCTOBER 25, 2020

Latest results from KRYSTAL-1 early clinical trial of adagrasib (MRTX849) Credit: Pasi A. Jänne New results from early clinical trials of a drug that targets a cancer-causing mutation in the KRAS gene have shown that it can shrink tumours and is well-tolerated by patients.

Gene 53

Gene Clinical Trials Clinical Trials Trials 53

XTalks

JUNE 18, 2024

In a field quickly gaining attention for its role in vaccine development and personalized medicine, Elixirgen Therapeutics stands out through its pursuit of innovation and safety. Founded in Baltimore, Maryland, a vibrant science hub, Elixirgen Therapeutics emerged from a vision to harness the untapped potential of mRNA technology.

Protein 52

Protein RNA Genetics Clinical Trials 52

Scienmag

JUNE 4, 2021

A new drug reduced tumor size in patients who have lung cancer patients with a specific, disease-causing change in the gene KRAS, a study found.

Genetics 65

Genetics Drugs Clinical Trials Clinical Trials 65