pharmaphorum

APRIL 23, 2021

billion in milestone payments in the deal, which will focus on using small-molecule compounds to “fine tune” the effects of genetic medicines in the body. billion in milestone payments in the deal, which will focus on using small-molecule compounds to “fine tune” the effects of genetic medicines in the body.

Gene Editing 52

Gene Editing Genetics Drugs Gene 52

Drug Discovery World

DECEMBER 6, 2023

“We are impressed by the unique delivery and editing technologies that Eligo has developed, and we believe this will set the stage for Eligo to forge new paths in the application of in vivo gene-editing technologies.” The investment will fuel pre-IND and IND activities to achieve early human data readouts in a PhIb/IIa clinical trial.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

Worldwide Clinical Trials

NOVEMBER 27, 2023

Food and Drug Administration (FDA) announced its acceptance of the Biologics License Application (BLA) for exa-cel. sickle cell disease community and the millions around the globe who suffer from this genetic illness. sickle cell disease community and the millions around the globe who suffer from this genetic illness.

Gene Editing 190

Gene Editing Gene Gene Therapy In-Vivo 190

Pharmaceutical Technology

NOVEMBER 4, 2022

The staff in the US from the acquired business will be retained by Reaction to set up its presence in Hershey, which comprises in vitro and in vivo expertise. The staff in the US from the acquired business will be retained by Reaction to set up its presence in Hershey, which comprises in vitro and in vivo expertise. at closing.

In-Vivo 130

In-Vivo In-Vitro Genetics Drugs 130

Drug Discovery World

JANUARY 12, 2023

Oksana Sirenko looks at the advantages and limitations of using organoids from human cell sources in drug development and drug screening. . Altering one base pair can drastically change how the gene’s protein product functions, while mutations can lead to the development of a range of genetic disorders. .

Gene Editing 98

Gene Editing Gene Drugs In-Vivo 98

Drug Discovery World

JANUARY 5, 2024

In non-human primate studies, the therapy has demonstrated effective, durable, and well tolerated in vivo CAR-T cell generation. In non-human primate studies, the therapy has demonstrated effective, durable, and well tolerated in vivo CAR-T cell generation.

In-Vivo 52

In-Vivo Development Licensing Licensing 52

Drug Discovery World

MAY 9, 2023

Jantzen Sperry , PhD, Director of Scientific Operations at Certis Oncology looks at how the adoption of orthotopic PDX models can elevate the impact of cancer research and improve translation in oncology drug development. Fewer than 5% of oncology drugs that enter clinical trials in the US receive US FDA approval.

Development 52

Development Drugs In-Vivo Clinical Trials 52

Drug Discovery World

DECEMBER 12, 2022

The fifth CRISPR in Drug Discovery: From Targets to Therapeutics meeting will be held face to face In Oxford on February 7-8, 2023. . The event will aim to provide a current perspective of the applications of these rapidly developing technologies with an emphasis on drug discovery applications. . Register here. Register here.

Genome 52

Genome Genomics Engineer In-Vivo 52

Drug Discovery World

APRIL 24, 2024

The strategic partnership will combine Variant Bio’s genomic discovery capabilities and VB-Inference platform with Evotec’s knowledge of antifibrotic drug discovery. The strategic partnership will combine Variant Bio’s genomic discovery capabilities and VB-Inference platform with Evotec’s knowledge of antifibrotic drug discovery.

Development 52

Development In-Vivo In-Vitro Genome 52

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). Other companies – notably Vertex Pharma/CRISPR Therapeutics and bluebird bio – are taking an ex vivo approach to SCD and thalassaemia and are much closer to market.

Gene Editing 81

Gene Editing In-Vivo Gene Sequencing Gene 81

Drug Discovery World

MARCH 7, 2024

Ginkgo is also working with Sensible Biotechnologies to develop an in vivo microbial mRNA manufacturing platform. Additionally, Ginkgo announced the successful completion of a manufacturing optimisation program with Biogen, focused on redefining the industry standard for manufacturing recombinant adeno-associated virus (AAV)-based vectors.

Gene Therapy 52

Gene Therapy RNA Engineer Gene 52

Drug Discovery World

JANUARY 23, 2023

Researchers have discovered a clinical drug that sensitises glioblastoma (GBM) cells to radiotherapy and could replace the current standard of care. In addition, they discovered that cladribine, a clinical drug, activates DGKB and inhibits DGAT1. GBM is a WHO grade IV brain tumour with dismal prognosis.

FDA Approval 52

FDA Approval Drugs In-Vivo In-Vitro 52

Pharmaceutical Technology

SEPTEMBER 29, 2022

The alliance will leverage the CRISPR genome editing technologies of Scribe to facilitate in genetic modification of new natural killer (NK) cell therapeutics for cancer. Under the agreement, Sanofi will receive non-exclusive rights to Scribe’s CRISPR by Design platform of wholly-owned enzymes for developing ex vivo NK cell therapies.

Development 130

Development In-Vivo Genome Genomics 130

Drug Discovery World

DECEMBER 15, 2022

Gene editing tools such as zinc finger nucleases, transcription activator-like effector nucleases (TALEN) and clustered regularly interspaced short palindromic repeat (CRISPR) nucleases have been heralded for their enormous potential to treat diseases and genetic disorders. In one example, Choi et al.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

pharmaphorum

JANUARY 10, 2022

The German group is paying $40 million upfront to kick off the alliance, which is focusing on the development of up to five CRISPR drugs that can be delivered in vivo for liver-targeted diseases. – are around one-third the size of the more commonly used Cas9.

In-Vivo 52

In-Vivo Gene Editing Gene Therapy Gene 52

Pharmaceutical Technology

MAY 18, 2023

Takeda oncology drug discovery unit head Kathy Seidl stated: “We are encouraged by KSQ’s CRISPRomics platform and its ability to perform in vivo genetic screens for the discovery and validation of tumour targets, which have the potential to modulate the innate and adaptive immune system. “We

Gene Therapy 130

Gene Therapy In-Vivo Gene Genetics 130

Pharmaceutical Technology

MAY 24, 2023

The ability to modify or introduce genetic material in human cells in such a precise and patient-centered manner clearly constitutes a breakthrough in personalized medicine. CGT drugs fall into two major categories: autologous and allogeneic. CGT drugs fall into two major categories: autologous and allogeneic.

Clinical Trials 246

Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain 246

Drug Discovery World

FEBRUARY 22, 2024

Lu Rahman looks at neuroscience drug discovery – advances and challenges – and how breakthroughs in this field are helping address areas of unmet need. Recent advances in biomarkers, research into the central nervous system and breakthroughs in human iPSC cell models, have bolstered neuroscience drug discovery research. to £104.7m

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59

Drug Discovery World

JUNE 30, 2023

Research tools, safety testing and regenerative medicines – these endless stem cell applications are powering precision, speed, and new modalities in drug development. Stem cells for use in drug discovery Stem cells are fast becoming an invaluable tool in the drug discovery process.

Development 52

Development Drugs In-Vitro Gene Therapy 52

Drug Discovery World

AUGUST 7, 2023

Together, Evox and Icahn Mount Sinai are focused on tackling a long-standing challenge in cardiovascular medicine, namely the safe and effective delivery of genetic medicines to cardiomyocytes.

Gene Therapy 52

Gene Therapy Gene Research In-Vivo 52

Drug Discovery World

JUNE 8, 2023

sAD has no specific family link and is caused by a combination of genetics, the environment and a person’s lifestyle. sAD has no specific family link and is caused by a combination of genetics, the environment and a person’s lifestyle. The recent US Federal Food and Drug Administration Agency (FDA) Modernization Act 2.0

Clinical Trials 97

Clinical Trials Clinical Trials Trials In-Vitro 97

WCG Clinical

MAY 28, 2024

CAR-T Cells Target Harmful B Cells in Lupus CAR-T cell technology, which uses genetic engineering to direct white blood cells to attack specific molecular targets, was originally proposed for treatment of HIV infection and hematological malignancies. The currently approved CD-19 CAR products are autologous, ex vivo cellular therapies.

In-Vivo 52

In-Vivo Clinical Trials Clinical Trials FDA Approval 52

Drug Discovery World

AUGUST 15, 2023

Can potentially lethal arrhythmias induced by novel drugs be better predicted by using more relevant and sophisticated electrophysiological screening methods? Such techniques, if routinely deployed in drug discovery, may enable improved decision making in predicting serious adverse cardiovascular events.

Drugs 52

Drugs In-Vivo Development Research 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

APRIL 12, 2021

Sanofi has licensed a potential inflammatory diseases drug from Manchester University spin-off C4X Discovery Holdings in a deal worth up to €414 million ($492 million). While the headline figure looks significant the UK biotech will receive only €7 million up front, reflecting the fact that the drug has not yet begun clinical testing.

Drugs 52

Drugs In-Vivo Licensing Licensing 52

Drug Discovery World

NOVEMBER 15, 2022

Anita Ramanathan outlines four synthetic biology approaches that are improving and accelerating drug discovery. . It has made its way into almost every aspect of drug discovery, whether it’s exploring basic biology, modelling diseases, identifying and validating targets, or performing drug screens. .

DNA 98

DNA Drugs Antibody Engineer 98

Scienmag

AUGUST 5, 2020

GW Cancer Center researchers demonstrate for the first time that HDAC6 inhibitors drugs can both improve response to immunotherapy and diminish the invasiveness of breast cancer WASHINGTON (Aug.

In-Vivo 41

In-Vivo Genetics Drugs Research 41

World of DTC Marketing

JANUARY 31, 2021

Porter points out that R&D seems to have been replaced with mergers and acquisitions, but she misses the big picture by focusing solely on drug costs. So where to start… Congresswoman Porter may want to look at the percentage of generic drugs available virus branded (89%). In pharma R&D returns have declined to 1.8

In-Vivo 187

In-Vivo Generic Drugs Gene Therapy Drugs 187

Pharmaceutical Technology

MARCH 9, 2023

Innovation S-curve for the pharmaceutical industry Transgenic murine models is a key innovation area in pharmaceutical s Transgenic murine models refer to mice that have been genetically altered for the purposes of understanding the in vivo functions of genes.

In-Vivo 130

In-Vivo Antibody Genetics Gene 130

Drug Discovery World

DECEMBER 12, 2023

I look forward to having access to this therapy as quickly as possible.” I look forward to having access to this therapy as quickly as possible.” The post RNA editing oligonucleotide selected for AATD treatment appeared first on Drug Discovery World (DDW).

RNA 52

RNA In-Vivo Genotype Genetics 52

Drug Discovery World

MARCH 20, 2024

Around 30% of drugs show adverse reactions during human clinical trials despite promising pre-clinical studies. One of the major causes of the high attrition rate is the poor predictive value of current preclinical models used in drug development. billion for bringing a new drug to market, a process extending over a decade.

Research 59

Research Clinical Trials Clinical Trials In-Vivo 59

pharmaphorum

JANUARY 29, 2021

Since that discovery, a flurry of gene-editing focused biopharma companies have launched – including Intellia Therapeutics, CRISPR Therapeutics, Caribou Biosciences and Mammoth Biosciences – and the first drug therapies based on the technology are now in human testing for diseases like cancer. Zinc fingers. billion in funding.

Gene Editing 142

Gene Editing Gene Gene Therapy In-Vivo 142

Drug Discovery World

APRIL 4, 2023

The Food and Drug Administration (FDA) in the US has made several key drug decisions over the last few weeks. The drug was granted orphan drug designation by the FDA in May 2021. The drug is expected to become a standard of care for these patients, who have had limited treatment options, according to analysts GlobalData.

FDA Approval 52

FDA Approval In-Vivo Gene Editing Drugs 52

Drug Discovery World

JUNE 19, 2023

Anoat will use the funds to conduct additional in vivo and in vitro pharmacology assessments and select a lead candidate. Cystic fibrosis is a progressive genetic disease that affects the lungs, pancreas and other organs.

Development 52

Development Life Science In-Vivo In-Vitro 52

Drug Discovery World

FEBRUARY 15, 2024

The etherna paradigm for cLNP design is ushering in the rapid generation of novel families of delivery systems for not only vaccination but also for in vivo tolerisation for a host of autoimmune diseases.” The post LNP formulations produce strong immune responses, data shows appeared first on Drug Discovery World (DDW).

Immune Response 52

Immune Response In-Vivo Vaccination Vaccine 52

Drug Discovery World

SEPTEMBER 14, 2023

Business updates Pfizer reported second quarter 2023 revenues totalling $12.7 billion, a decrease of $15.0 billion, or 54%, compared to the prior year quarter, reflecting an operational decline of $14.7 Vaccines were up 9.1%

Drugs 59

Drugs Vaccination Vaccine Medicine 59

pharmaphorum

OCTOBER 6, 2022

These include exposure to various chemicals associated with the war, from chemical warfare agents to pyridostigmine bromide – a preventative drug given to soldiers at risk of exposure to toxic chemicals. Disease modelling Gulf War Illness. In the mid-to-late 1990s, an unexplained illness began to emerge in army bases across the US.

In-Vivo 98

In-Vivo Research Genetics Drugs 98