STAT News

NOVEMBER 4, 2022

A man with muscular dystrophy who was first in line to receive an experimental gene editing therapy tailor made to treat the cause of his rare form of the disease has died. Terry had long been too old to participate in clinical trials of experimental therapies for the disease, which are often geared towards young boys.

Gene Editing 105

Gene Editing Gene Gene Therapy Scientist 105

pharmaphorum

FEBRUARY 3, 2021

Dr Jennifer Harbottle, senior scientist in the R&D Base Editing team of PerkinElmer’s Horizon Discovery business, looks at progress made in the realms of biotechnology and next-generation diagnostics, vaccines and therapeutics, including the application of CRISPR-Cas9 gene editing in developing and refining cell therapies.

Gene Editing 98

Gene Editing Gene DNA Engineer 98

pharmaphorum

OCTOBER 7, 2020

Drs Emmanuelle Charpentier and Jennifer Doudna have won this year’s Nobel Prize for chemistry in recognition of their work on the gene-editing technology CRISPR/Cas9. The technique introduces a break in a specific place within DNA that triggers a self-repair mechanism. million) Nobel Prize award. “In

Gene Editing 105

Gene Editing Bacteria DNA Gene 105

pharmaphorum

FEBRUARY 19, 2021

Once someone is infected with HIV it stays with them for life thanks to the retrovirus’s ability to inject its DNA code into the host. Excision wants to take its lead therapy candidate, EBT-101, into a phase 1/2 clinical trial in patients with chronic HIV infection with the proceeds from this latest financing round.

Gene Editing 105

Gene Editing In-Vivo DNA In-Vitro 105

pharmaphorum

APRIL 23, 2021

CRISPR Therapeutics is to receive a hefty $900m payment from Vertex after the companies amended a collaboration to develop, manufacture and market a gene editing therapy for sickle cell disease and beta thalassemia.

Gene Editing 64

Gene Editing Gene DNA Bacteria 64

Pharmaceutical Technology

MARCH 1, 2023

Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. The drug candidate targets DNA-dependent protein kinase (DNA-PK). The drug candidate is administered through the oral route in the form of capsule and tablet. It is a new chemical entity.

Gene Editing 100

Gene Editing DNA Cosmetics Life Science 100

Pharmaceutical Technology

MARCH 1, 2023

Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. The drug candidate targets DNA-dependent protein kinase (DNA-PK). The drug candidate is administered through the oral route in the form of capsule and tablet. It is a new chemical entity.

Gene Editing 100

Gene Editing DNA Cosmetics Life Science 100

XTalks

DECEMBER 20, 2023

This includes analyzing how drug combinations may impact individual patients or groups of patients before even entering a clinical trial. With a staggering 90 percent of drugs failing in clinical trials, AI has the potential to help improve these statistics. These companies are at various stages of research and clinical trials.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

pharmaphorum

AUGUST 31, 2022

In recent trials, researchers found that when the gene therapy was injected into the spinal cords of mice with ALS that SynCav1 protected and preserved spinal cord motor neurons and extended the lifespan of the mice. The list of companies supported includes Moderna, a company focused on mRNA that has also branched into gene editing.

Gene Therapy 98

Gene Therapy Gene Gene Editing DNA 98

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. Clinical trials have demonstrated the potential of CRISPR/Cas9 in this field.

Research 118

Research Gene Editing Gene Genetics 118

XTalks

NOVEMBER 30, 2023

The safety and efficacy of Cabenuva were established through two randomized, open-label, controlled clinical trials involving 1,182 HIV-infected adults who were virologically suppressed (HIV-1 RNA less than 50 copies/mL) before initiating Cabenuva treatment. The Phase I trial for HB-500 is scheduled to start in the first half of 2024.

FDA Approval 111

FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

XTalks

MAY 22, 2023

Related: 5 Food Companies Working With Precision Fermentation Technology How Pairwise Leverages CRISPR Technology CRISPR, or Clustered Regularly Interspaced Short Palindromic Repeats, is a gene-editing technique that can be used to alter the DNA of cells to enhance certain characteristics or reduce less desirable ones.

Branding 98

Branding Gene Editing DNA Production 98

pharmaphorum

APRIL 20, 2022

The potential of CRISPR technology has been a hot topic in the industry ever since it was first developed, but as trials progress further into the clinic, what therapeutic areas could be set to benefit? What this means is that it can edit virtually any segment of DNA, whilst being more precise and cheaper than existing editing tools.

Gene Editing 52

Gene Editing DNA Gene Genetics 52

XTalks

MAY 25, 2023

The review incorporated 283 studies, including randomized controlled trials and observational studies. CRISPR, or Clustered Regularly Interspaced Short Palindromic Repeats, is a gene-editing technique that can be used to alter the DNA of cells to enhance certain characteristics or reduce less desirable ones.

Gene Editing 52

Gene Editing Production DNA Gene 52

Delveinsight

JANUARY 14, 2021

The companies schedule to initiate a Phase 2 trial assessing combination therapy for both treatment-experienced and treatment-naïve people living with HBV. People in the trial with HBV treatment experience may also receive Gilead’s Vemlidy (tenofovir alafenamide fumarate, TAF). Gilead Sciences, Inc. and Vir Biotechnology, Inc.

Gene Editing 40

Gene Editing Gene Genetics DNA 40

XTalks

DECEMBER 13, 2023

The CRISPR system is dubbed to be molecular “scissors” that can cut out parts of genes that are abnormal, deactivating or replacing them with new strands of normal DNA. This has tremendous potential for curing diseases that are caused by faulty/mutated genes. Both therapies will be available in early 2024.

Gene Therapy 98

Gene Therapy Gene FDA Approval In-Vivo 98

The Pharma Data

NOVEMBER 22, 2021

It’ll support AstraZeneca’s focus on specialised and perfection drugs and foster the discovery and development of coming generation rectifiers, including nucleotide- grounded, gene-editing and cell curatives. region in the US, and Gothenburg in Sweden. Of the Company’s workers, further than work simply in R&D.

Gene Editing 52

Gene Editing Engineer Production Scientist 52

XTalks

DECEMBER 22, 2021

The gene-editing tool CRISPR edits DNA using specialized strands of guide RNA and endogenous cellular mechanisms of RNA interference. Double-stranded RNA is flagged by cells as foreign, triggering the cell’s RNA interference machinery to dice it up and destroy it.

Life Science 98

Life Science RNA Vaccine Vaccination 98

The Pharma Data

DECEMBER 27, 2020

This year has been difficult, but it has reinforced the importance of our mission: to treat and potentially even reverse the effects of serious diseases and conditions by advancing our novel cell therapy product candidates through clinical trials and into the hands of physicians.

Manufacturing 40

Manufacturing Production Development Clinical Trials 40

XTalks

AUGUST 2, 2023

This is largely due to failed trials as a prophylaxis resulting in the European Committee for Medical Products (CHMP) recommending against the approval of Lagevrio. There was a record 34 approvals in major markets, and late-stage trials are commencing for medications including camizestrant, datopotamab deruxtecan and volrustomig.

Sales 98

Sales Manufacturing FDA Approval Life Science 98

XTalks

DECEMBER 24, 2020

From rare disease drug approvals to treatments involving immunotherapies and gene therapies and awarding of a Nobel Prize to the inventors of the gene-editing tool CRISPR, 2020 was a year of great activity and productivity despite the backdrop of the pandemic. CRISPR Gene Editing Inventors Win Nobel Prize.

Life Science 111

Life Science Vaccine Vaccination Gene 111

Pharmaceutical Technology

FEBRUARY 20, 2023

If clinical trial participants are representative of the wider disease population, they can help sponsors assess the effectiveness of new and existing therapeutics. of the clinical trial population globally. participation of white subjects. The technology platforms are almost the same.

Research 130

Research Clinical Trials Clinical Trials Trials 130