XTalks

MARCH 7, 2024

In a pivotal move for the food industry, the US Food and Drug Administration (FDA) has unveiled industry guidance for genome edited foods derived from plants. The FDA’s recent guidance document sheds light on the regulatory landscape for foods derived from genome edited plants.

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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. The post AstraZeneca pays record 660% premium for gene editing company LogicBio appeared first on.

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pharmaphorum

OCTOBER 13, 2022

Gene therapies and research into them have grown immensely in recent years, offering more novel tools in regenerative medicine to fight disease, including rare diseases and genetic disorders. Beta-thalassemia is a rare blood disorder caused by a genetic defect in hemoglobin. Novartis also recently inked an up-to $1.5

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Gene Editing Gene Gene Therapy Genetics 52

pharmaphorum

APRIL 23, 2021

Vertex Pharma has ramped up its involvement in gene-editing medicines for the second time in a week, paying Obsidian Therapeutics $75 million upfront to access its technology platform. . Alternatively, a drug can be administered that switches off the genetic medicine and brings protein expression to a halt.

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Drug Discovery World

FEBRUARY 16, 2023

Challenges remain with current delivery approaches, and genetic medicines applications have been generally restricted to the liver and ex vivo settings. The post Start-up aims to advance transformative genetic medicines appeared first on Drug Discovery World (DDW).

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pharmaphorum

OCTOBER 8, 2020

Scribe Therapeutics, a start-up focusing on gene-editing using CRISPR/Cas9, has burst onto the biotech scene with a $415 million deal with Biogen. Its main platform – called X-editing – can generate CRISPR drugs with improved activity, specificity and deliverability compared to rivals, according to the start-up’s website.

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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.

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Drug Discovery World

MARCH 25, 2024

Allogene Therapeutics and Arbor Biotechnologies Biotechnology companies Allogene Therapeutics and Arbor Biotechnologies entered a non-exclusive, global gene editing licensing agreement for use of Arbor’s proprietary CRISPR gene-editing technology in Allogene’s AlloCAR T platform for the treatment of autoimmune disease (AID).

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Gene Editing Drugs Genome Genomics 52

XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

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Drug Discovery World

OCTOBER 6, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

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Drug Discovery World

JUNE 29, 2023

This 24-page eBook sponsored by The Jackson Laboratory looks at the key use-cases for genomics and how it is improving drug discovery and development projects.

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Genome Genomics Gene Editing Drugs 52

Drug Discovery World

OCTOBER 10, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

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Gene Editing Genome Genomics Genetics 59

Drug Discovery World

JUNE 29, 2023

The latest eBook by DDW ‘The impact of genomics on drug discovery: Creating new opportunities’ is out now. This 24-page eBook sponsored by The Jackson Laboratory looks at the key use-cases for genomics and how it is improving drug discovery and development projects.

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Genome Genomics Gene Editing Drugs 52

Worldwide Clinical Trials

OCTOBER 24, 2023

While this is a small number of people in each rare disease patient community, the significant and continued growth of rare disease drug development is unsurprising given that there are over 10,000 rare diseases , with more being discovered every year. Roughly 80% of rare diseases are due to a known genetic driver.

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pharmaphorum

JUNE 23, 2022

Novartis has shouldered its way into the in vivo gene editing category via a deal with US biotech Precision BioSciences, focused on a therapy for sickle cell disease (SCD). It is also working with Eli Lilly on in vivo gene-editing drugs against three targets, including one in Duchenne muscular dystrophy, under the terms of a $1.4

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Drug Discovery World

OCTOBER 25, 2022

Scientists have shown how stomach cancers can develop resistance to a new class of drugs called ATR inhibitors by switching off the activity of two key genes – raising the possibility of outsmarting cancer by predicting drug resistance in advance. . CRISPR used to identify genes involved.

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Development Drugs Gene Editing Gene 52

Pharmaceutical Technology

MARCH 2, 2023

According to GlobalData, Phase I drugs for Relapsed Acute Myeloid Leukemia have a 71% phase transition success rate (PTSR) indication benchmark for progressing into Phase II. GlobalData’s report assesses how QN-023a’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks.

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Gene Editing Genetic Engineering Engineer Clinical Trials 100

Drug Discovery World

OCTOBER 10, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

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Gene Editing Genome Genomics Genetics 52

Pharmaceutical Technology

MARCH 2, 2023

According to GlobalData, Phase I drugs for B-Cell Acute Lymphocytic Leukemia (B-Cell Acute Lymphoblastic Leukaemia) have a 90% phase transition success rate (PTSR) indication benchmark for progressing into Phase II. The company develops gene-editing organ transplantation technology. Buy the report here.

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Gene Editing Clinical Trials Clinical Trials Genetics 100

Drug Discovery World

OCTOBER 10, 2023

The National Institutes of Health has awarded the first phase of a roughly $40m grant to Yale University to advance a novel CRISPR-based gene-editing delivery platform for the targeted treatment of neurogenetic diseases. The two diseases being targeted in the project are Angelman syndrome and H1-4 (HIST1H1E) syndrome.

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Gene Editing Development Drugs Clinical Trials 52

Drug Discovery World

MAY 24, 2023

This exceeds the current, but growing, global supply of plasmid DNA for genetic medicine, and dramatically increases Touchlight’s capacity to meet growing demand for DNA from biopharmaceutical companies. The post New London facility exceeds global plasmid DNA demand appeared first on Drug Discovery World (DDW).

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DNA Gene Editing Gene Therapy Genetics 52

Drug Discovery World

JANUARY 17, 2023

Oregon State University College of Pharmacy scientists have shown that lipid nanoparticles and messenger RNA could treat blindness associated with a rare genetic condition. . We used these peptides to act as zip codes to deliver nanoparticles carrying genetic materials to the intended address within the eye.” .

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Gene Editing Gene Gene Therapy Genetics 98

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. Continue to STAT+ to read the full story…

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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. Overcoming drug resistance: A major challenge in cancer treatment is the development of resistance to chemotherapy drugs.

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Drug Discovery World

JUNE 22, 2023

“This is part of our long-term business strategy of leveraging exosome-mediated delivery to create and enable genetic medicines such as gene therapy and genome editors”. The post Evox Therapeutics acquires exosome AAV technology from Codiak appeared first on Drug Discovery World (DDW).

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Gene Therapy Gene Editing Drug Delivery Gene 52

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.

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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.

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Gene Editing Gene Genetic Engineering DNA 52

Drug Discovery World

JUNE 7, 2023

The disease-relevant mutation is engineered into bit.bio’s human ioWild Type  cells using CRISPR/Cas9 gene editing. This enables the manufacture of any human cell type at scale with precision and consistency, making the models suited to screening applications for early drug discovery as well as for fundamental research.

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Drug Discovery World

FEBRUARY 28, 2024

Lu Rahman selects some of the year’s interesting and noteworthy advances in cancer research drug discovery and development. As always, a year within cancer drug discovery and development brings with it significant breakthroughs, and 2023 has been no exception. Research into new cancer drugs remain strong. expressing cancers.

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Development Research Drugs Antibody 65

pharmaphorum

FEBRUARY 16, 2022

She received a cord blood transplant to treat the AML, receiving stem cells carrying a genetic mutation (CCR?5/?32) Having a third functional cure of HIV nevertheless does give pointers to how a more widely applicable cure might be developed in the future, for example using gene-editing drugs to introduce CCR5?32

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Gene Editing Research Gene Genetics 69

Drug Discovery World

NOVEMBER 14, 2023

FH is one of the most common genetic conditions, affecting around one in 300 people globally. It is caused by a single gene mutation that impairs the body’s ability to mediate LDL cholesterol. Since the mutation only affects a single gene, it makes it a prime candidate for genome editing treatments.

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Drug Discovery World

NOVEMBER 16, 2023

Casgevy (exagamglogene autotemcel, or exa-cel) is the first medicine to be licensed that uses gene editing tool CRISPR, for which its inventors were awarded the Nobel Prize in 2020. 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.

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Drug Discovery World

APRIL 24, 2023

A Phase III clinical trial led by physicians in the US has shown that adding investigational drug motixafortide significantly increases the number of stem cells that can be harvested compared with treatment with the standard agent alone. The study also found that the combination worked rapidly and was generally well-tolerated by patients.

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Drugs Gene Editing Gene Genetics 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. There is enormous power in this genetic tool, which affects us all. What started as a curiosity?driven,

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Drug Discovery World

MAY 9, 2024

The post New cell engineering partnership to accelerate biopharma innovation appeared first on Drug Discovery World (DDW). The hTSC platform is the earliest ethically sourced pluripotent stem cells that are naive, powerful, lively and have high plasticity.

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Drug Discovery World

DECEMBER 12, 2022

DDW highlights the most interesting global news and comments within the drug discovery and development sector this month. . Everyone enjoys a good news story and the drug discovery and development sector is no exception. The post The Dose: DDW’s drug discovery highlights appeared first on Drug Discovery World (DDW).

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Drugs Gene Editing Genome Genomics 52

pharmaphorum

FEBRUARY 17, 2022

Global Blood Therapeutics’ run of positive regulatory news for its oral sickle cell disease treatment Oxbryta has continued with an approval for the drug in Europe for patients aged over 12. The post GBT claims EU approval for sickle cell drug Oxbryta appeared first on.

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