Chemical Biology and Drug Design

JULY 26, 2023

Exosome engineering can be conducted through surface and content modification of biological exosomes. A huge number of studies have mentioned the significant role of ncRNAs in cancer prevention and therapy that make them an interesting subject for cancer therapy. In postisolation method, some contents (e.g.,

Engineering 100

Engineering Therapies Cell Biology Drugs 100

Broad Institute

JANUARY 8, 2024

A team of researchers at the Broad Institute of MIT and Harvard has engineered virus-like particles to deliver prime editors to cells in mice at a high enough efficiency to rescue a genetic disorder. However, delivering the complex gene editing machinery to cells in living animals has been challenging.

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Engineering Research Virus DNA 144

Broad Institute

JUNE 27, 2024

Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain. Credit: Neumann EN, Bertozzi TM, et al.

Disease 142

Disease Therapies Engineering Clinical Trials 142

Broad Institute

JANUARY 14, 2025

Building on previous work by the vector-engineering lab of Ben Deverman at the Broad, the team developed a pair of adeno-associated viruses (AAVs) to package and deliver the base-editing machinery to brain cells. Theres still a long way to go to make this a therapy, Minikel said. But its really exciting to see how much is possible.

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Disease DNA Protein Production Treatment 144

Drug Target Review

AUGUST 28, 2024

Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years. Could you describe the platform of macrophage biology and cell engineering used by Resolution Therapeutics in developing their cell therapies?

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Therapies Disease Engineering Medical Schools 116

Codon

FEBRUARY 13, 2024

Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Read it on our website here. Illustration by David S. Fast forward to 2023.

Therapies 131

Therapies Virus DNA Clinical Trials 131

Drug Target Review

NOVEMBER 8, 2023

Problem w/ CTs and foundational understanding of Vittoria: can you explore the current limitations of cell therapies and the challenges faced by patients and providers? Currently, only a small percentage of cancers can be effectively treated with cell therapies, and there is little diversity in the currently approved products.

Therapies 116

Therapies Engineering Treatment Molecular Biology 116

Drug Target Review

JUNE 27, 2023

This ebook delves into the intricate world of cancer therapies, immune system breakthroughs, therapeutic genetic engineering, and groundbreaking advancements in gene editing, featuring insightful interviews with renowned experts in the field.

Therapies 105

Therapies Immune Response Engineering Drugs 105

Broad Institute

FEBRUARY 27, 2025

An ancient RNA-guided system could simplify delivery of gene editing therapies By Corie Lok February 27, 2025 Breadcrumb Home An ancient RNA-guided system could simplify delivery of gene editing therapies The programmable proteins are compact, modular, and can be directed to modify DNA in human cells. Lisa Yang and Hock E.

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RNA Therapies DNA Virus 66

Drug Target Review

JULY 5, 2023

Induced pluripotent stem cell (iPSC) therapies have the potential to treat a wide variety of devastating diseases. iPSC-derived lymphocytes, eg, T cells and natural killer (NK) cells, engineered to express targeting molecules such as chimeric antigen receptors (CARs) have shown clinical promise to treat haematological malignancies.

Therapies 103

Therapies DNA Engineering Virus 103

Science Daily: Pharmacology News

JUNE 26, 2023

Researchers have engineered a tissue-mimicking hydrogel model to show that more elastic tissues induce T cells to become effector-like T cells with strong tumor-killing potential, while more viscous tissues induce them to become memory-like T cells. T cells experience different mechanical signals in different tissues.

Engineering 195

Engineering Therapies Research 195

Drug Discovery Today

AUGUST 13, 2020

CPI, an independent technology innovation centre and founding member of the UK Government’s High Value Manufacturing Catapult, has today announced a partnership with Perceptive Engineering and FUJIFILM Diosynth Biotechnologies.

Engineering 100

Engineering Therapies 100

Drug Target Review

NOVEMBER 29, 2023

Our primary focus is to design and develop RNA therapies for liver diseases. Our team of chemists then uses this information to develop novel therapies. By doing this, we get a superior understanding of which therapies will and will not work in human biology before a clinical trial.

Therapies 102

Therapies Disease RNA Engineering 102

Drug Target Review

JULY 3, 2023

Dr David Baram from gene therapy company EmendoBio provides a snapshot of the history of therapeutic genetic engineering, explaining the early pitfalls and reasons for recent renewed optimism.

Therapies 98

Therapies Clinical Trials DNA Engineering 98

Broad Institute

JULY 19, 2023

Researchers reprogram gene therapy viral vectors to bind specific protein targets By Allessandra DiCorato July 19, 2023 Breadcrumb Home Researchers reprogram gene therapy viral vectors to bind specific protein targets A new screening method zeroes in on adeno-associated viruses that enter the brain through a defined mechanism.

Therapies 98

Therapies Research Engineering Virus 98

Drug Target Review

OCTOBER 2, 2024

Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. However, cell-based therapies are intended to leverage those healthy cells via transplant to regenerate damaged tissue.

Therapies 98

Therapies Disease Engineering RNA 98

Drug Target Review

SEPTEMBER 1, 2023

Could you explain the fundamental distinction between autologous and allogeneic cell therapies and how they differ in terms of cell sourcing and application? Autologous immune cell therapy involves the grafting of a patient’s own immune cells. Then, cells are infused back to that same patient.” There are pros and cons to each.

Therapies 105

Therapies Engineering Production Clinical Trials 105

Drug Target Review

NOVEMBER 17, 2023

They do not induce ‘graft versus host’ disease when transplanted for allogeneic therapy and there appears little sign of immune rejection. Peripheral blood NK cells have been used in allogeneic cancer therapies and shown to be safe. developing immune stem cell therapies for treating ovarian and gastric cancer.

Therapies 118

Therapies Clinical Trials Cell Biology Trials 118

Drug Target Review

JUNE 6, 2023

We are in an era of immuno-oncology (IO) revolution with many approved therapies now available to treat a broad range of cancers. For the last several years, the field has worked to unlock the potential of IO therapies for additional tumour types, and has explored options beyond checkpoint inhibitors.

Engineering 98

Engineering Immune Response Biochemical Pharmacology Clinical Trials 98

Broad Institute

AUGUST 7, 2024

Machine learning approach helps researchers design better gene-delivery vehicles for gene therapy By Allessandra DiCorato August 7, 2024 Breadcrumb Home Machine learning approach helps researchers design better gene-delivery vehicles for gene therapy The method could accelerate the development of more effective adeno-associated viruses (AAVs).

Therapies 84

Therapies Research Engineering Packaging 84

DrugBank

JUNE 13, 2024

These microscopic particles, which are smaller than 100 nanometers, can be engineered to deliver drugs to specific targets with precision.   Gene Therapy: Reprogramming the Body's Cellular Code    Gene therapy is an exciting field that treats diseases at their genetic roots.

Therapies 98

Therapies Drugs DNA Treatment 98

Covalent Modifiers

AUGUST 14, 2024

Rullo Molecular Therapy 2024 DOI: [link] Proximity-induction of cell-cell interactions via small molecules represents an emerging field in basic and translational sciences. We evaluated the utility of CIRs to direct anti-tumor function of human T cells engineered with three biologically distinct classes of SAR. Nickolas J.

Small Molecule 182

Small Molecule Engineering Science Therapies 182

Broad Institute

JUNE 10, 2024

The advance, from the lab of Broad core institute member David Liu , could one day help researchers develop a single gene therapy for diseases such as cystic fibrosis that are caused by one of hundreds or thousands of different mutations in a gene.

Engineering 144

Engineering DNA Therapies Disease 144

Drug Target Review

FEBRUARY 28, 2024

Over the past 25 years, T-cell therapies have gained significant ground in the treatment of cancer. Preclinical research on γδ T cells has made great strides since the cells were first identified in the 1980s, with γδ T-cell therapies from several companies, including IN8bio, now in or nearing clinical trials for various cancers.

Therapies 105

Therapies Engineering Molecular Biology Science 105

Drug Target Review

MAY 14, 2024

What potential therapeutic advantages do the engineered cationic polypeptides offer in the context of generating antitumour immune responses? One major obstacle of current immune checkpoint blockade therapies is to treat ‘solid tumours’ as immunosuppressive factors are highly activated within tumour microenvironments.

Engineering 59

Engineering Immune Response FDA Approval Clinical Trials 59

Drug Target Review

DECEMBER 13, 2024

2 Unmet needs in lung cancer treatment Recent decades have seen significant advancements in lung cancer treatment, especially with the introduction of targeted therapies and immunotherapies, which have notably improved survival rates. These can include anti-tubulin compounds, potentially reducing side effects and improving quality of life.

Therapies 98

Therapies Treatment Clinical Trials Drugs 98

Drug Target Review

DECEMBER 20, 2024

These twins simulate how a patients condition might evolve without treatment, enabling researchers to compare the real-world effects of an experimental therapy against predicted outcomes. Previously, Aaron was an algorithm engineer and manager of the hand tracking team at Leap Motion. Aaron received his Ph.D.

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Clinical Trials Trials Pharmaceuticals Drug Development 128

Drug Discovery Today

MARCH 8, 2021

Diverse panel of novel target binders from IONTAS / FJB will be used to uncover functional antibodies to advance Quell’s engineered Treg cell therapy pipeline

Engineering 113

Engineering Therapies 113

Drug Target Review

OCTOBER 26, 2023

Genome engineering and gene therapies that manipulate DNA sequences in cells have driven a biotechnological revolution over the past decade. The limitations of natural AAV serotypes in meeting all clinical needs has spurred growing interest in AI-driven AAV engineering approaches.

Engineering 105

Engineering Virus Therapies Immune Response 105

Drug Target Review

OCTOBER 6, 2023

This interview explores how CoRegen’s innovative techniques are not only transforming cancer treatment but also hold promise for a wide range of medical applications, all while minimising the side effects often associated with traditional therapies. What are the key benefits of your approach?

Small Molecule 111

Small Molecule Engineering Protein Expression Therapies 111

Covalent Modifiers

JUNE 24, 2024

Rullo, Molecular Therapy: Oncology , 2024 , 200842, [link] Proximity-induction of cell-cell interactions via small molecules represents an emerging field in basic and translational sciences. We evaluated the utility of CIRs to direct anti-tumor function of human T cells engineered with three biologically distinct classes of SAR.

Small Molecule 130

Small Molecule Engineering Science Therapies 130

Advarra

JUNE 12, 2023

Gene therapy research is exciting and full of promise, but because of the risks involved, it’s also highly regulated, requiring an institutional biosafety committee (IBC) to provide additional oversight and risk assessment. What Does an IBC Review? What Does an IBC Review?

Engineering 98

Engineering Therapies Regulations Research 98

Advarra

MARCH 31, 2023

The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. A key part of the IBC’s evaluation is assessing the risks posed by the engineered genetic materials.

Engineering 52

Engineering Clinical Research Research Virus 52

Broad Institute

JULY 10, 2024

The new method, published today in Nature Biomedical Engineering , precisely and durably corrects the mutation in human lung cells, restoring cell function to levels similar to that of Trikafta. Nature Biomedical Engineering. The most common is a three base-pair CTT deletion that causes the ion channel protein to misfold and degrade.

Engineering 145

Engineering Packaging DNA Treatment 145

Advarra

JUNE 13, 2024

Genetically engineered products often require additional safety practices to ensure the infectious agents do not endanger participants, study staff, or the broader environment where such agents are administered. The post The Importance of Hazard Communications in Clinical Trials Involving Genetic Engineering appeared first on Advarra.

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Clinical Trials Engineering Trials Virus 52

Drug Discovery Today

APRIL 22, 2020

Cambridge, UK, and Brisbane, CA, 21 April 2020 Mogrify Ltd (Mogrify®), a UK company aiming to transform the development of cell therapies by the systematic discovery of novel cell conversions, and Sangamo Therapeutics (Sangamo) (Nasdaq: SGMO), a genomic medicine company, today announced that they have executed a collaboration and exclusive license (..)

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Licensing Licensing Engineering Therapies 100

Drug Target Review

AUGUST 29, 2024

What potential advantages does the CD5 modulation strategy offer over traditional CAR-T therapies? MR : Chimeric antigen receptor T-cell (CAR-T) therapy is very effective in treating patients with B-cell lymphoma, leukemia, and multiple myeloma, where we have six FDA-approved drugs.

Therapies 59

Therapies Treatment Engineering Molecular Biology 59