LifeSciVC

JUNE 12, 2025

Founding Vision: Harnessing RNA Editing for Transformative Medicine Korro Bio’s story began in 2018, co-founded by a remarkable group: Jean-François Formela, M.D. Academic specializing in RNA editing). The ability of modifying RNA enabled us to sidestep many of the potential risks associated with permanent DNA editing.

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RNA Protein Expression DNA Science 67

Drug Target Review

JULY 5, 2023

Induced pluripotent stem cell (iPSC) therapies have the potential to treat a wide variety of devastating diseases. While iPSC therapies hold great promise, several challenges remain, including poor infiltration of cytotoxic lymphocytes into solid tumours and insufficient cytotoxicity of myeloid cells.

Therapies 103

Therapies DNA Engineering Virus 103

Fierce BioTech

NOVEMBER 1, 2023

Plasmid DNA Design Considerations for Cell and Gene Therapy Cell and Gene therapy organizations must balance the need for rapid clinical progression and stringent quality expectations throughout development.

DNA 52

DNA Therapies RNA 52

Broad Institute

JANUARY 8, 2024

In the new work published today in Nature Biotechnology , the team adapted engineered virus-like particles (eVLPs) that they had previously designed to carry base editors — another type of precision gene editor that makes single-letter changes in DNA. Nature Biotechnology. Online January 8, 2024.

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Engineering Research Virus DNA 144

Covalent Modifiers

AUGUST 14, 2023

Acquired cysteines are both driver mutations and sites targeted by precision therapies. For both cancer and healthy genomes, we find that cysteine acquisition is a ubiquitous consequence of genetic variation that is further elevated in the context of decreased DNA repair.

Small Molecule 130

Small Molecule RNA DNA Therapies 130

Broad Institute

OCTOBER 23, 2024

In recent years, genetic editing technologies and other gene therapy approaches have given scientists the ability to alter the genes inside living cells. That is in part because of the ongoing challenge of understanding the DNA switches, called cis-regulatory elements (CREs), that control the expression and repression of genes.

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Research DNA Laboratories Molecular Biology 136

Drug Target Review

JULY 11, 2025

Xenium takes this further with in situ analysis at subcellular resolution, detecting RNA and protein molecules directly within tissues using high-resolution imaging. From discovery to therapy Schnall-Levin believes the industry is edging closer to real-world therapies that will be directly traceable to single-cell and spatial analysis. “I

Molecular Biology 69

Molecular Biology Disease Therapies Bioinformatics 69

Drug Target Review

JULY 7, 2023

The reasons for this are multifaceted, including concerns over the safety of directly altering DNA sequences and subsequent regulatory restrictions that have arisen as a result. The epigenome (meaning ‘above the genome’) is a system of reversible marks regulating how the DNA is read, translated and used. What is epigenetic editing?

DNA 105

DNA Engineering Therapies Regulations 105

Broad Institute

OCTOBER 23, 2024

By Leah Eisenstadt, Broad Communications October 23, 2024 Credit: Courtesy of the Broadbent family Brian and Julia Broadbent are raising their daughters Claire, top left, and Emma, seated, who is the first person to be diagnosed with a rare genetic disorder caused by the long noncoding RNA CHASERR.

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DNA RNA Disease Hospitals 128

DrugBank

JANUARY 29, 2025

Gene Therapy Gene therapy operates on the principle of modulating the DNA blueprint of cells to induce a therapeutic response. Gene therapy trials explore using lentiviral vectors to deliver a functional copy of the HBB gene to hematopoietic stem cells, the progenitors of all blood cells.

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Therapies DNA RNA Disease 52

PLOS: DNA Science

MARCH 28, 2024

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. But even if gene therapy could reach sufficient muscle cells to improve quality of life, another hurdle looms: the dystrophin gene that’s deleted in DMD is huge, the largest in a human genome at 2.2

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FDA Approval Treatment FDA DNA 94

Alta Sciences

APRIL 16, 2024

Inside The Altascientist: The Benefits of PCR for Your Gene Therapy Programs pmjackson Tue, 04/16/2024 - 19:04 The gene therapy landscape continues to accelerate in preclinical and clinical research, with programs constantly in development for targeted, personalized medicines. But how are PCR analysis techniques applied?

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Therapies DNA RNA Laboratories 52

Drug Target Review

SEPTEMBER 28, 2023

After some time in that role and launching several products, I received a call from Bill Banyai and Bill Peck, or ‘The Bills’ as we call them, who were building a company around technology that creates DNA by ‘writing’ it on a silicon chip. Researchers were able to use our synthetic RNA controls as a reference to verify and validate assays.

DNA 116

DNA Organic Chemistry Science Virus 116

DrugBank

MARCH 12, 2025

Introduction Messenger RNA (mRNA) technology has emerged as one of the most significant medical breakthroughs.   The Expanding Role of mRNA in Cancer Therapy One of the most exciting applications of mRNA therapeutics lies in cancer treatment, where leveraging the immune system to target tumors offers a novel approach.

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Vaccine Treatment Clinical Trials Therapies 97

Alta Sciences

FEBRUARY 19, 2025

The guidance ensures that these targeted evaluations help identify and mitigate safety concerns early in the development process, supporting the safe progression of oligonucleotide therapies into clinical trials. An oligonucleotide is a short strand of nucleotides, the building blocks of DNA or RNA, used in genetic research and therapy.

Drug Development 52

Drug Development Clinical Pharmacology Immune Response Drugs 52

Broad Institute

MAY 14, 2025

Cas9 ancestor engineered into a compact genome editing tool By Tom Ulrich May 14, 2025 Breadcrumb Home Cas9 ancestor engineered into a compact genome editing tool Researchers adapt a compact RNA-guided enzyme from bacteria for a variety of DNA editing tasks in human cells.

Engineering 59

Engineering DNA RNA Therapies 59

Drug Target Review

NOVEMBER 13, 2023

The instructions to form a cell are encoded in DNA strands, wrapped in proteins and RNA, and wound together into a 3D structure called chromatin. Another finding was that regulatory T cells had distinct chromatin architecture features called DNA loops. This indicated other Foxp3 protein-containing complexes may be involved.

Government 104

Government DNA RNA Regulations 104

Codon

JUNE 11, 2024

And unlike traditional DNA sequencers, which parse genetic material by breaking it up into fragments and interpreting it chunk-by-chunk, a nanopore device unspools a long strand of DNA and reads it all at once. A scientist can isolate DNA and load up a flow cell in fifteen minutes. Nanopore devices work incredibly fast.

DNA 99

DNA Engineering RNA Small Molecule 99

Broad Institute

DECEMBER 13, 2023

The team analyzed the full transcriptome of cells from nearly 100 regions across the mouse brain using high-throughput single-nucleus RNA sequencing, the preferred approach for efforts to create a human brain atlas. Related news Researchers map brain cell changes in Alzheimer’s disease Courtesy of the Chen and Macosko labs.

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DNA Virus Research Engineering 140

DrugBank

JULY 24, 2024

For example, PROTACs targeting STAT3, a protein crucial for tumor growth, have shown promise where conventional therapies have failed. The versatility and potential of PROTACs are vast, paving the way for new therapies that could transform how we treat diseases.

Small Molecule 98

Small Molecule Drugs Clinical Trials Therapies 98

Fierce BioTech

NOVEMBER 16, 2023

Beyond cfDNA: Harnessing the Power of Exosomes to Improve Sensitivity for Cancer Mutation Detection Liquid biopsy markers are advantageous because they can be used to assess overall prognosis, response to therapy, and likelihood of relapse or progression. Download now. Download now.

RNA 52

RNA DNA Therapies Treatment 52

DrugBank

APRIL 24, 2025

Epigenetics , the study of changes in gene activity that occur without altering the DNA sequence, has revolutionized our understanding of gene expression regulation. These therapies aim to reverse abnormal epigenetic patterns, restore normal gene function, and offer hope for previously uncurable diseases.

DNA 52

DNA Drugs Therapies Regulations 52

The Pharma Data

NOVEMBER 17, 2021

Mount Sinai researchers have developed a new model that uses DNA and RNA sequencing data from hundreds of patients to identify specific genes and genetic alterations responsible for never-before-defined subtypes of a blood cancer called multiple myeloma.

Treatment 52

Treatment RNA DNA Science 52

Alta Sciences

MARCH 1, 2024

Norbert Makori and Pierre Jolicoeur: The Benefits of Combining Preclinical and Bioanalytical for Your Gene Therapy Studies pmjackson Fri, 03/01/2024 - 22:20 Recent advances in gene therapy have allowed for diseases to be approached differently. q: How can Altasciences support sponsors’ Gene Therapy Programs?

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Therapies Laboratories Molecular Biology RNA 52

The Pharma Data

FEBRUARY 22, 2022

State-of-the-art facility will headquarter research efforts in Boston and New York to accelerate the development of RNA and DNA-based medicines.

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RNA DNA Disease Therapies 52

Conversations in Drug Development Trends

AUGUST 14, 2024

Hematological cancer cells can also develop resistance to therapies over time, reducing treatment effectiveness. Fortunately, personalized treatment plans help circumvent therapy resistance while optimizing response rate and improving quality of life.

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Treatment Therapies Disease Protein Expression 78

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

DrugBank

MAY 15, 2024

Another promising avenue is the use of technologies like RNA interference and gene editing, which allow scientists to turn off the production of certain proteins altogether. 5 This period also saw the rise of biologics in the 1980s, which have played a crucial role in modern therapies, particularly for diseases like cancer.

Small Molecule 98

Small Molecule Molecular Biology Drugs Disease 98

The Pharma Data

OCTOBER 21, 2020

In the midst of the global pandemic, the analysis found no antiretroviral therapy interruptions across the entirety of the ongoing clinical development programme for long-acting cabotegravir and rilpivirine. Of those participants who transitioned back to injectables, the median duration of oral therapy was 51 days.

Clinical Development 52

Clinical Development Therapies Clinical Trials Treatment 52

Dark Matter Blog

JULY 16, 2021

As soon as I learned about DNA and RNA, I wanted to be a molecular biologist. Last stops at RNA My last roles in biotech were where my original passion began: DNA and RNA. My last stop at Arrakis Therapeutics is with a company targeting RNA with small molecules. Arrakis is the capstone of my career.

Small Molecule 52

Small Molecule RNA Clinical Trials DNA 52

Drug Target Review

APRIL 3, 2024

Everything started in school with an experiment on isolating DNA from bananas. Making a little tangle of DNA visible to the eye and understanding that this is the basis of complex organisms, which might be altered in disease, was the defining moment for my future path in life sciences. in North America) in late 2017.

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Science Clinical Development Treatment DNA 105

Drug Target Review

APRIL 9, 2024

In a previous study, we showed that these peptides decorate all cellular RNAs and thus impair the binding of RNA-binding proteins to RNA. For his postdoctoral stay he joined the group of André Nussenzweig, where he started to work on DNA repair, particularly focusing on the role of histone H2AX.

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RNA Cell Biology Clinical Development DNA 64

The Pharma Data

OCTOBER 19, 2020

. “Innovation in cancer treatment is realized through collaboration, and the physicians and researchers at Emory’s Winship Cancer Institute have consistently shown their commitment to developing novel cancer therapies using this collaborative approach,” said Chadi Nabhan , M.D., Ramalingam , M.D.,

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Clinical Trials RNA DNA Science 52

The Open Targets Blog

AUGUST 15, 2023

Research in mice has shown that RBM3 expression is protective against misfolded protein damage, suggesting the protein as an interesting target in the development of therapies for neurodegenerative conditions such as Alzheimer’s disease. Curiously, these followed a theme in molecular function: the regulation of splicing*.

RNA 52

RNA Treatment Regulations DNA 52

The Pharma Data

NOVEMBER 22, 2020

Back in September, Vertex Pharmaceuticals and CRISPR Therapeutics – the company behind the CRISPR Cas-9 platform – announced that the European Medicines Agency (EMA) had granted Priority Medicines (PRIME) designation to CTX001, an investigational ex vivo CRISPR Cas-9 gene-edited therapy for the treatment of severe sickle cell disease.

DNA 52

DNA Medical Schools Science RNA 52

Vial

DECEMBER 5, 2023

In 2017, the Food & Drug Administration (FDA) approved the first gene therapy for cancer and for inherited diseases, the first multiplex NGS panel for companion diagnostics (CDx), and the first drug targeting a genetic signature though not a disease. Fountzilas et al.

Clinical Trials 52

Clinical Trials Trials RNA Bioinformatics 52

KIF1A

FEBRUARY 4, 2024

We each have two DNA copies of the KIF1A gene; each copy is called an allele and makes its own RNA, which is then translated into protein. It is a type of gene-based therapy for KIF1A mutations that reduces RNA expression of the mutant allele, without interrupting healthy KIF1A expression. What is an allele specific ASO?

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RNA DNA Disease Trials 52