Science Daily: Pharmacology News

JULY 5, 2023

An antisense therapy restores production of the protein FMRP in cell samples taken from patients with fragile X syndrome.

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Protein Production Production Therapies RNA 189

BioPharma Drive: Drug Pricing

JUNE 3, 2024

The alliance hands Lilly an RNA-based therapy in preclinical testing for ALS and frontotemporal dementia, and includes a research collaboration to develop other, similar medicines.

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RNA Therapies Drugs Research 218

Science Daily: Pharmacology News

JULY 27, 2023

If translated into the clinic, this approach could expand access and reduce the cost of gene therapies for blood disorders, many of which currently require patients receive chemotherapy and a stem cell transplant.

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RNA Research Therapies Disease 183

BioPharma Drive: Drug Pricing

JANUARY 17, 2024

The biotech is developing an RNA-based therapy for what it describes as a root cause of the pregnancy-related complication, which affects millions of women.

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RNA Therapies Drugs 201

BioPharma Drive: Drug Pricing

FEBRUARY 22, 2024

The $260 million fund will be used to back startups working on new drugmaking approaches like protein degraders and RNA therapies.

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RNA Therapies 184

BioPharma Drive: Drug Pricing

NOVEMBER 12, 2024

Trace Neuroscience aims to advance an RNA-binding therapy designed to preserve — and potentially improve — muscle function in people living with the nerve-destroying disorder.

RNA 167

RNA Therapies 167

Drug Target Review

JUNE 15, 2023

In this article Drug Target Review’s Izzy Wood spoke to Sam Hasson, Director of Target Biology at Rgenta Therapeutics, a biotech firm in Massachusetts, US, that aims to develop small molecule therapeutics to target RNA processing. The field is faced with a number of obstacles that require specifically assay technologies to surmount.

RNA 96

RNA Small Molecule Disease Assay Development 96

Broad Institute

MAY 16, 2024

By Allessandra DiCorato May 16, 2024 Credit: Deverman lab Brain vasculature (in blue) surrounded by RNA (in orange) transcribed from the gene delivered to the brain in humanized mice using an engineered AAV targeting the human transferrin receptor.

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Therapies FDA Approval Engineering Disease 137

BioPharma Drive: Drug Pricing

JUNE 3, 2024

With new modalities such as gene and cell therapies, RNA therapeutics, complex biologics and more, today’s science brings unprecedented opportunities to address diseases that have long remained out of reach.

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Science RNA Therapies Disease 144

Drug Target Review

JANUARY 2, 2024

Through a series of experiments, they successfully developed a personalised stem cell therapy using a data-driven, single-cell technique based on swift subcellular proteomic imaging. Our recent work proposes the use of an intracellular toolkit to map organelle bio-geography in stem cells that could lead to more precise therapies.”

RNA 128

RNA Immune Response Therapies Disease 128

Drug Target Review

APRIL 7, 2025

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. These fields explore highly precise biological processes related to RNA, specific proteins and gene expression mechanisms.

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Clinical Development Clinical Trials Clinical Pharmacology Trials 69

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.

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Therapies DNA Engineering Virus 103

Elrig

MARCH 25, 2025

This second ELRIG meeting on Therapeutic Oligonucleotides brings together esteemed scientists from academia, industry, and other members of the drug discovery community to explore the discovery, validation, and targeting of oligonucleotide-based drug candidates, including antisense oligonucleotides (ASOs) and small interfering RNA (siRNA).

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RNA Bioinformatics Science Pharmacokinetics 59

Drug Target Review

DECEMBER 6, 2024

This includes verifying the experimental design and understanding how the data was generated whether it was from RNA sequencing, mass spectrometry, or other biological assays. Figure 1: High-level workflow for early drug discovery Once the raw data has been gathered, the next step is to gain a thorough understanding of the data.

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RNA Drugs Research Science 52

Covalent Modifiers

AUGUST 14, 2023

Acquired cysteines are both driver mutations and sites targeted by precision therapies. Our chemoproteogenomics platform integrates chemoproteomic, whole exome, and RNA-seq data, with a customized 2-stage false discovery rate (FDR) error controlled proteomic search, further enhanced with a user-friendly FragPipe interface.

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Small Molecule RNA DNA Therapies 130

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.

Therapies 52

Therapies DNA RNA Disease 52

Fierce BioTech

APRIL 10, 2025

Listing Image QuartzListingLogo_250x190 10x Genomics.png Listing Introduction Join us for an exclusive webinar exploring groundbreaking findings from the DUTRENEO trial, which evaluated the role of predictive biomarkers in guiding neoadjuvant therapy for muscle-invasive bladder cancer (MIBC).

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Clinical Trials RNA Trials Therapies 52

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

Now the researchers describe how they re-engineered both eVLPs and parts of the prime editing protein and RNA machinery to boost editing efficiency up to 170 times in human cells compared to the previous eVLPs that deliver base editors. DOI: 10.1038/s41587-023-02078-y Tags: Liu Lab Gene editing Rare Disease Gene-based therapy delivery

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Engineering Research Virus DNA 144

Chemical Biology and Drug Design

AUGUST 24, 2023

Abstract Targeted therapy has attracted more and more attention in cancer treatment in recent years. However, due to the diversity of tumor types and the mutation of target sites on the tumor surface, some existing targets are no longer suitable for tumor therapy.

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RNA Therapies Drugs Treatment 100

Drug Target Review

JULY 11, 2024

To date, there are less than 30 known surface proteins that form the basis of all approved cancer-targeted therapies of all modalities, including antibody-drug conjugates (ADCs), T-cell engagers, CAR T-cells and radiopharmaceuticals. These could be targets for antibody-drug conjugates (ADCs), bi-specific antibodies, or other therapies.

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Therapies RNA Pharma Companies Disease 56

ASPET

OCTOBER 8, 2024

Finally, RNA sequencing revealed a potential link in the downregulation of Ras/MAPK signaling following combination treatment. Significance Statement Increasing evidence suggests that oxidative phosphorylation might play a critical role in the development of resistance to cancer therapy.

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RNA Treatment Therapies Disease 100

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

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

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

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.

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Drug Development Clinical Pharmacology Immune Response Drugs 52

Chemical Biology and Drug Design

FEBRUARY 26, 2024

The m 6 A-RNA immunoprecipitation (MeRIP) assay detected the m 6 A modification profile. In all, METTL3 induced miR-4654 maturation in a m 6 A-dependent manner, which was then reduced SOD2 expression, thus promoting apoptosis and oxidative stress in HLECs, suggesting a novel path for DC therapy.

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RNA Regulations Therapies Research 100

Crown Bioscience

JULY 15, 2024

The rapid increase of precision oncology approaches in recent years has been fueled in part by technological innovations and an increased interest in personalized therapies.

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RNA Therapies Treatment Disease 52

Drug Target Review

JULY 7, 2023

This gives epigenetic editing the unique ability to treat complex multigenic diseases and opens the door to enhancing cell and regenerative therapies, which require the manipulation of multiple genes for optimal results. She is currently focused on using genetic tuning to enhance and develop cell and gene therapy products for clinical impact.

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DNA Engineering Therapies Regulations 105

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. Like Cas9, IscB enzymes cut DNA at sites specified by an RNA guide.

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Engineering DNA RNA Therapies 59

The Pharma Data

MARCH 9, 2022

Novartis today announced a license option agreement with Voyager Therapeutics, a gene therapy company focused on next-generation adeno-associated virus (AAV) technologies, for three capsids to use in potential gene therapies for neurological diseases, with options to access capsids for two other targets.

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Licensing Licensing Therapies Disease 52

Drug Target Review

SEPTEMBER 25, 2024

Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.

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Small Molecule Therapies Regulations Treatment 105

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.

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Small Molecule Drugs Clinical Trials Therapies 98

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. This limits our ability to design gene therapies that only effect certain cell types in the human body."

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Research DNA Laboratories Molecular Biology 136

Broad Institute

NOVEMBER 7, 2023

While RA therapies targeted to specific inflammatory pathways have emerged, only some patients’ symptoms improve with treatment, emphasizing the need for multiple treatment approaches tailored to different disease subtypes.

Treatment 96

Treatment RNA Hospitals Disease 96

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

Broad Institute

JANUARY 22, 2024

As part of the Cancer Program at the Broad, the Sellers lab explores the link between genetic alterations and cancer dependencies with the goal of informing new therapies. Initially, there were two approaches: small hairpin RNA (shRNA)-based and now CRISPR-based experiments. There are a number of limitations to those approaches.

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Drugs Therapies Regulations RNA 128