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. The post Nobel winner Charpentier’s CRISPR Therapeutics gets $900m in reworked Vertex deal appeared first on.

Gene Editing 64

Gene Editing Gene DNA Bacteria 64

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. It also affects Hispanic Americans, but at a lower prevalence.

Gene Therapy 95

Gene Therapy Gene FDA Approval In-Vivo 95

Pharmaceutical Technology

MARCH 1, 2023

According to GlobalData, Phase I drugs for Neuroendocrine Tumors have a 74% phase transition success rate (PTSR) indication benchmark for progressing into Phase II. GlobalData’s report assesses how Nedisertib’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks.

Gene Editing 100

Gene Editing DNA Cosmetics Life Science 100

Pharmaceutical Technology

MARCH 1, 2023

According to GlobalData, Phase I drugs for Head And Neck Cancer Squamous Cell Carcinoma have an 80% phase transition success rate (PTSR) indication benchmark for progressing into Phase II. GlobalData’s report assesses how Nedisertib’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks.

Gene Editing 100

Gene Editing DNA Cosmetics Life Science 100

pharmaphorum

MAY 7, 2021

Gene editing firm CRISPR Therapeutics has announced a collaboration with US biotech Nkarta to develop natural killer (NK) cell therapies for cancer. It’s at the forefront of gene editing although the technology has spawned rivals including Intellia Therapeutics, Caribou Biosciences, Sangamo Therapeutics and Mammoth Biosciences.

Gene Editing 52

Gene Editing Gene Genetic Engineering DNA 52

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.

Gene Editing 105

Gene Editing Bacteria DNA Gene 105

STAT News

SEPTEMBER 16, 2022

Enjoy, and see you soon … Intellia Therapeutics reported that the first six patients to receive its CRISPR-based treatment for a genetic swelling disorder have safely had small, corrective changes made to dysfunctional DNA inside their liver cells , STAT writes.

Gene Editing 94

Gene Editing Genetic Disease Genetics Protein 94

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 109

Research Gene Editing Gene Genetics 109

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. The cash will also help develop two other preclinical drugs, EBT-104 for Herpes Simplex Virus and EBT-107 for hepatitis B.

Gene Editing 105

Gene Editing In-Vivo DNA In-Vitro 105

The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Selim Corbacioglu from the University of Regensburg, relies on CRISPR-Cas9 gene editing to switch off BCL11A. SATURDAY, Dec.

Gene Therapy 52

Gene Therapy Gene Gene Editing Genetics 52

XTalks

NOVEMBER 30, 2023

In 2022, the US Food and Drug Administration (FDA) granted approval to the medication Sunlenca (lenacapavir) for adults living with multi-drug resistant HIV-1 infection. Developed by Gilead, Sunlenca is the first drug in a novel class known as capsid inhibitors to receive FDA approval for treating HIV-1.

FDA Approval 110

FDA Approval Gene Therapy Gene Clinical Trials 110

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 91

Branding Gene Editing DNA Production 91

Cloudbyz

MARCH 6, 2024

Traditionally, pharmaceutical therapies have primarily consisted of small molecule drugs, commonly known as pills. This shift signifies not just a change in the way drugs are manufactured and administered, but also a fundamental transformation in the approach to treating diseases at their root cause.

Gene 99

Gene Immune Response Manufacturing Gene Therapy 99

Delveinsight

FEBRUARY 25, 2021

Beam adds to drug delivery stable with USD 120 Million GuideTx buy. The gene-editing technologies, which have reached the clinic, CRISPR, zinc finger nucleases, and TALENs, make their edits by nicking DNA at the target site. If they eliminated the T cells, the therapy stopped working.

DNA 52

DNA In-Vivo Genetics Medicine 52

XTalks

MAY 12, 2021

In this episode, Ayesha discusses a new tool that uses CRISPR to modulate gene expression without editing DNA sequences. Dubbed “CRISPRoff,” the technology targets the epigenome to silence genes involved in diseases, with applications in cancer, AML and other conditions with a heritable component.

Life Science 52

Life Science Gene Expression Gene DNA 52

Delveinsight

MARCH 2, 2021

HitGen & UPPTHERA Collaborate for Drug Delivery Research. HitGen has entered into a collaboration for drug discovery research with UPPTHERA to identify small molecule Hits against previous known-to-be undruggable targets and novel E3 ligase. Artiva Raises USD 120 M to Advancing Allogeneic NK Cell Therapy Pipeline.

Drug Delivery 40

Drug Delivery Research Drugs Gene Editing 40

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

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. The treatment used a new version of CRISPR to turn on a backup copy of the dystrophin gene that mysteriously lies dormant in our muscles.

Gene Editing 105

Gene Editing Gene Gene Therapy Scientist 105

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. The new£ 1bn installation will include the most advanced robotics, high- outturn webbing and AI- driven technology.

Gene Editing 52

Gene Editing Engineer Production Scientist 52

XTalks

DECEMBER 20, 2023

As we step into 2024, the life sciences continue to evolve at an unprecedented pace, driven by technological innovation, a deeper understanding of human biology and the application of new technologies in areas like drug development and health wearables. Regulatory bodies are also taking note of the applications of AI in drug development.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

pharmaphorum

JULY 13, 2022

Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment. dosing of *first patient* with VERVE-101, an in vivo CRISPR base editing medicine. Today: we are announcing.

Gene Editing 98

Gene Editing Trials Drugs In-Vivo 98

pharmaphorum

APRIL 20, 2022

In 2012, Jennifer Doudna and Emmanuelle Charpentier published a paper in Science where they outlined isolating the components of the CRISPR-Cas9 system and demonstrated how it could be used to cut specific sites in isolated DNA. The publication and their work eventually led to the pair being awarded the Nobel Prize in Chemistry in 2020.

Gene Editing 52

Gene Editing DNA Gene Genetics 52

The Pharma Data

DECEMBER 2, 2020

Food and Drug Administration (FDA) for Onivyde (irinotecan liposome injection) for study patients with small cell lung cancer (SCLC) who progressed following a first-line platinum-based regimen. Results show the protocol demonstrated a 30Gb+ yield of long DNA reads raw data of an E. The drug is a small-molecule inhibitor of the S.

Drugs 52

Drugs Gene Editing Genome Genomics 52

XTalks

AUGUST 2, 2023

Pfizer’s Top 5 Best-Selling Drugs of 2022: 1) Comirnaty Comirnaty is an mRNA-based vaccine indicated for the prevention of COVID-19. Comirnaty was first approved by the US Food and Drug Administration (FDA) in August 2021 for individuals over the age of 16. billion the drug generated in 2021. billion in 2022. billion, a 26.55

Sales 91

Sales Manufacturing FDA Approval Life Science 91

XTalks

DECEMBER 22, 2021

At the beginning of the year, Genentech, a member of the Roche Group, struck a deal with Ribometrix to leverage its discovery platform to discover, develop and commercialize small molecule drug candidates against RNA targets. The deal could bring the North Carolina-based RNA-based small molecule therapeutics developer more than $1 billion.

Life Science 95

Life Science RNA Vaccination Vaccine 95

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. Drugs, Testing and Vaccines. Drug Approvals.

Life Science 108

Life Science Vaccination Vaccine Gene 108

Pharmaceutical Technology

MAY 26, 2023

Enzymatic DNA production company Touchlight have augmented its DNA production capabilities with a newly announced expansion to its London facilities. Tripling its production capacity, Touchlight can now manufacture 8kg plasmid DNA, a key component for mRNA gene therapies and vaccines.

DNA 130

DNA Manufacturing Gene Gene Therapy 130

Pharmaceutical Technology

FEBRUARY 20, 2023

On the other hand, a lack of diversity in clinical trial populations, particularly when the disease population is significantly underrepresented, greatly diminishes the quality of data obtained for drug safety and efficacy profiles. This may sound obvious, but it is still a major hurdle in drug development.

Research 130

Research Clinical Trials Clinical Trials Trials 130