Broad Institute

OCTOBER 23, 2024

Machine learning guides researchers to new synthetic genetic switches By Tom Ulrich October 23, 2024 Breadcrumb Home Machine learning guides researchers to new synthetic genetic switches A new method allows precise activation or repression of genes in specific cells and tissues. By Sarah C.P.

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Drug Target Review

JANUARY 2, 2024

In research published in Scientific Reports , 1 investigators focused on mesenchymal stem cells (MSCs), known for their potential in treating cell defects and regulating immune responses. 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.”

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Drug Target Review

DECEMBER 6, 2024

In drug discovery and biological research, the scientists workflow often follows a structured and iterative approach to ensure accuracy, reproducibility and scientific integrity. At the heart of any research is the scientific question. This acts as a safeguard against false positives and misinterpretation of the data.

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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

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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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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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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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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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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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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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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The Pharma Data

JANUARY 19, 2021

A naturally occurring system for tuning CRISPR-Cas9 expressing in bacteria, identified in a study published in Cell , could have implications for gene editing therapies as well. A CRISPR-Cas9 system has two components: the Cas9 guide RNA that directs the system to edit a particular gene, and the CRISPR “scissors” that make the cut.

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Alta Sciences

FEBRUARY 19, 2025

Researchers must characterize the anti-drug-antibody (ADA) response in preclinical and clinical studies and report any ADA-positive samples as a risk-based approach. 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 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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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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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. They also found that patients’ CTAPs were dynamic and could change over time in response to treatment.

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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. Author Bio: Dr Blythe Sather Dr Blythe Sather is the Vice President, Head of Research at Tune Therapeutics.

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Fierce BioTech

MARCH 14, 2025

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). Don't miss this opportunity to gain deep insights from leading experts in oncology and biomarker research.

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Broad Institute

JANUARY 22, 2024

At the Broad Institute of MIT and Harvard and elsewhere, researchers are also learning how dependencies affect cancer cells and how they influence each other and contribute to drug resistance. How have researchers looked for cancer dependencies in the past? There are a number of limitations to those approaches.

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Broad Institute

AUGUST 19, 2024

A team of researchers at the Broad Institute of MIT and Harvard; the Ragon Institute of Mass General Brigham, MIT, and Harvard; and St. Jude Children’s Research Hospital have discovered that oleic acid, one of the most abundant fatty acids in the body, restores a healthy balance of vaginal microbes in a laboratory model of BV.

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Hospitals Treatment Medical Schools Science 121

Broad Institute

DECEMBER 13, 2023

By Leah Eisenstadt December 13, 2023 Two teams of Broad researchers produced detailed maps of the mouse nervous system. Related news Researchers map brain cell changes in Alzheimer’s disease Courtesy of the Chen and Macosko labs. Courtesy of the Chen and Macosko labs. Courtesy of the Chen and Macosko labs.

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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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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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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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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. Difficult-to-treat and rare, complicated cancers remain a perplexing challenge for IO researchers. Clinical development of IL-18 therapies has been curtailed, however, by the protein’s lack of efficacy.

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KIF1A

JULY 29, 2023

Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. Next week we’re overjoyed to convene families, clinicians, and researchers to discuss progress and goals in our mission to find treatments and cures of KAND.

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RNA Clinical Trials Disease Science 98

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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PLOS: DNA Science

MARCH 28, 2024

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. Research also took place in two facilities in Italy, with sponsorship from Italfarmaco S.p.A. Researchers have been working on developing gene therapy for DMD for decades. million DNA bases.

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DrugBank

MAY 15, 2024

Researchers would sift through natural extracts , hoping to stumble upon a compound that exhibited beneficial effects. In fact, it's one of the most exciting areas in drug research today. With billions in funding and innovative research, we're starting to see strategies that can tackle these tough targets.

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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. Biomarker data interpretation can be difficult, particularly for oncology studies.

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Elrig

DECEMBER 14, 2023

Federico Dajas-Bailador, an expert in axon biology, discusses groundbreaking research, unraveling neuronal function and the potential for treating neurological disorders. Learn how RNA's role in axons opens avenues for targeted therapies reshaping neurological condition treatments.

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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.

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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. Researchers have long understood that Foxp3 is essential for regulatory T cell development, but only as an on-off switch for regulatory T cell genes.

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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. ” The Institute will be headquartered in 334,000 sq. of leased space in a 12-story building, developed and operated by Alexandria Real Estate Equities, Inc.,

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Drug Target Review

AUGUST 24, 2023

Neutralising GDF-15 with CatalYm’s anti-GDF-15 antibody visugromab was shown to reverse its inhibitory effects and to re-sensitise tumours to anti-PD-1 treatment, achieving commensurate survival benefit of anti-GDF-15-anti-PD-1 combination therapy in vivo. Cancer Immunology Research. in North America) in late 2017.

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Drug Target Review

JULY 23, 2024

Shortly after finishing my studies, I landed my first job in industry working on cell biology research for several disease indications. At the time, research and leadership roles in biotech/pharma industry were predominately held by men and there were not as many opportunities for career development and mentorship from women leaders.

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LifeSciVC

JANUARY 16, 2024

These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline. TfR1 is almost becoming table stakes for Pharma and biotech looking to deliver to the brain and / or muscle.

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