Drug Discovery World

MAY 17, 2024

From a weight loss drug that prevents heart attacks and a gene therapy that restores hearing, to a vaccine that can treat viruses that don’t exist yet, our chosen news stories this week all represent potential breakthroughs in their respective fields. News round-up for 13-16 May by DDW Senior Digital Content Editor Diana Spencer.

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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.

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Gene Editing Genetics Drugs Gene 52

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. .

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Gene Editing Gene Drugs In-Vivo 98

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.

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Gene Editing Gene In-Vivo Genetics 52

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.

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In-Vivo In-Vitro Genetics Drugs 130

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.

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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. AdBio Partners’ mission is to support founding scientists in the development of new therapeutic approaches,” said Alain Huriez, Managing Partner of AdBio Partners. “We

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Development Life Science In-Vivo In-Vitro 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). billion in potential milestones if the project advances through development and onto the market. billion agreement that started in 2020.

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Gene Editing In-Vivo Gene Sequencing Gene 97

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.

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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.

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Life Science Gene Editing Development Clinical Trials 119

Pharmaceutical Technology

MAY 18, 2023

This partnership will focus on the identification and validation of new targets to inform treatment developments that address resistance mechanisms in cancer patients. KSQ Therapeutics has announced the expansion of its immuno-oncology strategic partnership with Takeda for new tumour-intrinsic targets.

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Gene Therapy In-Vivo Gene Genetics 130

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.

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Gene Therapy RNA Engineer Gene 52

XTalks

AUGUST 5, 2020

Single cell RNA sequencing technology has been extensively applied to understand heterogeneity among tumor cells, within the tumor microenvironment and during cell development. Single cell sequencing has revolutionized the study of biological tissues and systems at the cellular and molecular level. The Power of Single Cell Technology.

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Gene Expression Development RNA Drugs 112

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

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Clinical Trials Clinical Trials Trials In-Vitro 97

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

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Drugs Gene Clinical Trials Clinical Trials 59

pharmaphorum

JANUARY 10, 2022

Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences. There is another $1 billion in downstream payments on offer, assuming the drug candidates all make it through to regulatory approval and hit sales targets.

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In-Vivo Gene Editing Gene Therapy Gene 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.

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Research Clinical Trials Clinical Trials In-Vivo 59

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? We suggest a new generation of screening assays, that better recapitulate the electrophysiology of the whole human heart, may allow better decisions across research and development.

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Drugs In-Vivo Development Research 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.

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FDA Approval Drugs In-Vivo In-Vitro 52

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

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. .

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DNA Drugs Antibody Engineer 98

Drug Discovery World

AUGUST 7, 2023

The collaboration will work on developing exosome-encapsulated AAV (exoAAV) vectors as a novel gene delivery technology aimed at improving treatments for heart disease. Evox Therapeutics has agreed a research collaboration and option agreement with the Icahn School of Medicine at Mount Sinai (Icahn Mount Sinai) in New York, NY, US.

Gene Therapy 52

Gene Therapy Gene Research In-Vivo 52

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.

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Drugs In-Vivo Licensing Licensing 52

Drug Discovery World

DECEMBER 12, 2023

Selecting a development candidate for our AATD program is an important milestone for Korro, but more importantly, for patients. KRRO-110 is designed to co-opt an endogenous enzyme Adenosine Deaminase Acting on RNA (ADAR) to repair a pathogenic single nucleotide variant (SNV) on RNA and restore production of normal A1AT.

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RNA In-Vivo Genotype Genetics 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%

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Drugs Vaccination Vaccine Medicine 59

Drug Discovery World

APRIL 12, 2023

By DDW Editor Reece Armstrong A lot has already been said about the US Food and Drug Administration’s (FDA) Modernization Act 2.0 The decision by the Biden administration to end the federal requirement for drugs under development to be tested on animals represents a significant step forward for the life sciences sectors.

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In-Vivo Clinical Trials Clinical Trials Drugs 52

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.

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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. In these cases B cell depletion is a feature, not a bug.

In-Vivo 52

In-Vivo Clinical Trials Clinical Trials FDA Approval 52

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.

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Gene Editing Gene Gene Therapy In-Vivo 145

The Pharma Data

DECEMBER 13, 2020

which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., as Head of Business Development. .–( BUSINESS WIRE )– EdiGene, Inc. as Head of the US Subsidiary, and Kehua Fan, M.D.,

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pharmaphorum

OCTOBER 6, 2022

A complex range of factors are thought to contribute to the development of the illness. 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.

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In-Vivo Research Genetics Drugs 98

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.

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FDA Approval In-Vivo Gene Editing Drugs 52

Drug Discovery World

FEBRUARY 6, 2023

WM : Infinimmune is an early-stage antibody biotechnology company, focused on reinventing antibody drug discovery by developing human-derived antibody drugs. However, the limited number of drugs and targets demonstrates the need for new approaches to both discovery and development.

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Antibody Immune Response Genome Genomics 52

Drug Discovery World

FEBRUARY 15, 2024

Hypothetically, any mRNA-based drug using S-Ac7-DOG as the lipid base would therefore have improved efficacy and a better safety profile.

Immune Response 52

Immune Response In-Vivo Vaccination Vaccine 52

Drug Discovery World

AUGUST 14, 2023

Over the past few decades, the incredible progress in personalised medicine has led to the development of life-saving cell and gene therapies (CGT). Within the past year, the sector saw the EU’s landmark approval of the first allogeneic T cell therapy as well as the approval of several new gene therapies in the US. billion in 2020 to $15.5

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Gene Therapy Marketing Gene DNA 52

The Pharma Data

JANUARY 17, 2021

Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases. Those funds were used to advance its discovery and development programs.

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In-Vivo Marketing Engineer Genetics 52

Drug Discovery World

OCTOBER 27, 2023

Organ-chips combine cell culture with microfluidics to emulate the biological forces of different organ tissues and/or disease states, allowing pharmaceutical researchers to determine a drug candidates’ efficacy and toxicity ahead of clinical trials. Could organ-chips replace other research methods? Not yet, according to Ewart.

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Research Gene Therapy In-Vivo In-Vitro 72