Intellia cleared to start key CRISPR drug study in UK
Bio Pharma Dive
OCTOBER 19, 2020
The trial would be the first test of a systemically administered gene editing treatment that uses the Nobel Prize-winning technology to alter DNA.
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Bio Pharma Dive
OCTOBER 19, 2020
The trial would be the first test of a systemically administered gene editing treatment that uses the Nobel Prize-winning technology to alter DNA.
Bio Pharma Dive
SEPTEMBER 12, 2022
Scientists at Pretzel believe fixing mutated mitochondrial DNA with a mix of small molecule therapies and gene editing could be key to solving a number of hard-to-treat diseases.
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Bio Pharma Dive
NOVEMBER 14, 2022
The RNA drug developer is paying the richly-funded startup $80 million in a wide-ranging alliance that marks its first foray into DNA editing medicines.
STAT News
NOVEMBER 14, 2022
Ionis Pharmaceuticals, the California company that turned RNA-targeting medicines from an unproven idea into blockbuster drugs, is now expanding into a new class of therapeutics that treat disease by editing DNA. On Monday, the company announced a partnership with Metagenomi to kickstart these efforts.
Worldwide Clinical Trials
NOVEMBER 12, 2024
For example, once considered incurable and terminal, patients with sickle cell disease may reach new summits in their lives with gene editing technologies such as CRISPR to repair affected DNA and, in some cases, functionally cure the condition.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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. Overcoming drug resistance: A major challenge in cancer treatment is the development of resistance to chemotherapy drugs.
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.
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.
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.
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.
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. That would just slow the whole field down.
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.
Camargo
JULY 27, 2021
When developing a clinical development program for these increasingly popular therapies, it is important that sponsors use modeling and pharmacokinetic (PK) analysis to evaluate parameters that can be measured while dosing with gene therapy drugs, to characterize exposure-response data and inform rational dosing.
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.
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 clinical trial was to be the first time anyone got a gene editing therapy for muscular dystrophy.
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. Both therapies will be available in early 2024. million after discounts.
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.
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.
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.
The Pharma Data
DECEMBER 5, 2020
The other study, from German researchers led by Dr. Selim Corbacioglu from the University of Regensburg, relies on CRISPR-Cas9 gene editing to switch off BCL11A. The CRISPR gene editing method won the 2020 Nobel Prize in Chemistry, and uses chemicals to open up and directly edit DNA sequences, Hsu said.
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.
The Pharma Data
JUNE 7, 2023
Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., LNPs are spheric drug delivery bodies which can be equipped with therapeutic payloads for intracellular delivery. Financial details were not disclosed. Source link: [link]
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.
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.
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
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.
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.
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