Broad Institute

MAY 21, 2025

The score, called an MM-like score, assesses the severity of disease and risk of progression to active cancer, with higher scores indicating faster progression. The MM-like score defined in this study focuses on the presence and progressive evolution of genetic abnormalities to assess the state of the disease.

DNA 52

DNA Disease Bioinformatics Hospitals 52

Drug Target Review

JANUARY 8, 2024

It is no mystery that as we age our health starts to deteriorate, and we become increasingly susceptible to diseases. Through years of scientific exploration and research, we now know there are several biological changes that make our bodies more susceptible to disease or injury, and we can target those with therapeutic interventions.

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Science Disease FDA Approval Pharmaceutical Companies 145

LifeSciVC

JUNE 12, 2025

2025 holds to be a transformative year for us, with the potential for multiple milestones to come to fruition, with the biggest value inflection being the interim clinical data from our lead asset KRRO-110, a potential best in class compound for patients with Alpha-1 antitrypsin deficiency (AATD). Over the last 4.5

RNA 67

RNA Protein Expression DNA Science 67

New Drug Approvals

MAY 17, 2025

Retrieved 17 April 2025. ^ “Qalsody- tofersen injection” DailyMed. “FDA grants accelerated approval for Biogen ALS drug that treats rare form of the disease” CNBC. “FDA advisors vote against effectiveness of Biogen’s ALS drug for rare and aggressive form of the disease” CNBC.

FDA Approval 62

FDA Approval FDA Treatment Clinical Trials 62

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. Ten were capable of editing DNA in human cells.

Engineering 59

Engineering DNA RNA Therapies 59

Broad Institute

MARCH 27, 2025

Building bridges between Denmark-based and Broad researchers By Corie Lok March 27, 2025 Breadcrumb Home Building bridges between Denmark-based and Broad researchers The Novo Nordisk Foundation Center for Genomic Mechanisms of Disease is forging connections between Danish and Broad scientists. Here are two of their stories.

Research 52

Research DNA Regulations Disease 52

Broad Institute

JUNE 24, 2025

By Broad Communications June 24, 2025 Credit: Casey Atkins David Liu is a core member at the Broad Institute where he is the Richard Merkin Professor and director of the Merkin Institute for Transformative Technologies in Healthcare. s to be produced at scale, making them the standard of care for life-threatening rare genetic diseases.

Treatment 133

Treatment Disease DNA Clinical Trials 133

Broad Institute

JANUARY 14, 2025

By Allessandra DiCorato January 14, 2025 Credit: Maria Nemchuk, Broad Communications Eric Minikel and Sonia Vallabh run a lab with a singular focus: preventing and treating prion disease within their lifetime. Its a huge privilege to have the opportunity to point these powerful new tools at our disease.

Disease 144

Disease DNA Protein Production Treatment 144

Broad Institute

MAY 27, 2025

Gene editing disrupts Huntingtons mutation in mice By Allessandra DiCorato May 27, 2025 Breadcrumb Home Gene editing disrupts Huntingtons mutation in mice Making single-letter edits in stretches of repeated DNA stopped or reversed the genetic change that causes Huntingtons disease and Friedreichs ataxia.

DNA 133

DNA Disease Packaging Research 133

Codon

JUNE 5, 2025

Gray, in London to discuss the significance of her recovery at the Third International Summit on Human Genome Editing , described Casgevy as “a new beginning for people with sickle cell disease.” ” Despite its association with genome editing, 1 CRISPR didn’t start out as a tool for fighting genetic disease.

DNA 79

DNA RNA Clinical Trials Engineering 79

SugarCone Biotech

JULY 6, 2025

A post-ASCO update on tumor targets DLL3, B7H3 and HER3 July 2025 Paul D Rennert, SugarCone Biotech LLC Introduction: DLL3, B7H3 and HER3 are compelling tumor antigens to target with cancer therapeutics. However, HER3-DXd missed achieving a statistically significant improvement in overall survival, leading to the withdrawal of its U.S.

Drug Development 56

Drug Development Drugs Clinical Trials Therapies 56

Codon

JUNE 2, 2025

Art and technology often intersect in this way: a brush, a chisel, a 3D printer, DNA encoded with the image of a Germanic Rune , a biocompatible implant that induced the growth of an ear on a forearm, a bioprinter, CRISPR editing that induces pigmentation in bacteria. ” Asimov Press (2025). Cite: Nitkey S. Join Asimov Press.

Engineering 65

Engineering Therapies DNA Trials 65

Broad Institute

FEBRUARY 11, 2025

Mapping mRNA through its life cycle within a cell By Corie Lok February 11, 2025 Breadcrumb Home Mapping mRNA through its life cycle within a cell Xiao Wangs studies of how and where RNA is translated could lead to the development of better RNA therapeutics and vaccines.

RNA 52

RNA DNA Science Vaccine 52

Drug Target Review

DECEMBER 28, 2023

During the process of transformation from a normal cell into a cancer cell, a cell acquires a series of changes, or mutations, in its DNA. But DNA mutations can also result in changes to the proteins that are displayed on the surface of the cancer cell.

Bioinformatics 105

Bioinformatics Immune Response DNA Vaccine 105

Codon

SEPTEMBER 29, 2024

The earliest ones relied on simple linear regression and attempted to correlate genetic variations with observable traits or disease risks — such as drug metabolization rates or cancer susceptibility. The 2025 genetic network paper fit that bill exactly. 2024 Statistical models of organisms have existed for decades.

Engineering 144

Engineering Virus DNA Therapies 144

Codon

MAY 15, 2023

3/ Prime Editing Spree Prime editors can change DNA in ways that Cas9 — and even base editors — cannot. Known as a "search-and-replace" gene-editing tool, prime editors can delete or replace DNA up to 10,000 bases in length, or substitute one base for another. Read more in Cell. ( #1 , #2 ) (Video credit: Gong Y.

DNA 52

DNA Bioinformatics Engineering Virus 52

Codon

MAY 15, 2023

3/ Prime Editing Spree Prime editors can change DNA in ways that Cas9 — and even base editors — cannot. Known as a "search-and-replace" gene-editing tool, prime editors can delete or replace DNA up to 10,000 bases in length, or substitute one base for another. Read more in Cell. ( #1 , #2 ) (Video credit: Gong Y.

DNA 52

DNA Bioinformatics Engineering Virus 52

Broad Institute

MAY 29, 2025

New technique expands cells to sequence DNA and capture fine structural details By Allessandra DiCorato May 29, 2025 Breadcrumb Home New technique expands cells to sequence DNA and capture fine structural details Using this method, scientists discovered key protein and gene activity changes in aging and in a rare disease called progeria.

DNA 133

DNA RNA Cell Biology Disease 133

Broad Institute

MAY 15, 2025

Evolved gene editor inserts entire genes in human cells By Allessandra DiCorato May 15, 2025 Breadcrumb Home Evolved gene editor inserts entire genes in human cells The new system is the first to use a DNA-mobilizing enzyme called a CRISPR-associated transposase to make targeted gene-sized edits at therapeutically useful levels in human cells.

DNA 133

DNA Laboratories RNA Disease 133

Broad Institute

MARCH 6, 2025

Studies reveal new genetic roots of atrial fibrillation By Leah Eisenstadt March 6, 2025 Breadcrumb Home Studies reveal new genetic roots of atrial fibrillation Researchers double the number of genetic factors associated with this common arrhythmia, highlighting biological pathways that could be targeted by new medicines.

DNA 126

DNA Medical Schools Disease Therapies 126

Codon

MAY 8, 2025

If so, then here's a question: How much information is in your DNA? When I first looked into this question , I thought it was simple: Human DNA has about 3.1 Each base pair can take one of four values (A, T, C, or G) It takes 2 bits to encode one of four possible values (00, 01, 10, or 11) Thus, human DNA contains 6.2

DNA 75

DNA RNA Molecular Biology Regulations 75

Broad Institute

JANUARY 27, 2025

A genome-wide atlas of cell morphology reveals gene functions By Allessandra DiCorato January 27, 2025 Breadcrumb Home A genome-wide atlas of cell morphology reveals gene functions PERISCOPE, a technique for genome-wide imaging screens, is helping Broad scientists understand the connections between genes and traits. Online January 27, 2025.

DNA 131

DNA RNA Science Research 131

Codon

FEBRUARY 19, 2025

Researchers today often spend months trying to figure out whether a genetic mutation causes disease, simply because laboratory experiments are slow. The same model can also generate brand-new DNA sequences at the scale of yeast chromosomes or small bacterial genomes. Like ChatGPT, Evo 2 is a large language model.

DNA 105

DNA Laboratories Engineering RNA 105

Broad Institute

MAY 12, 2025

By Allessandra DiCorato May 12, 2025 Credit: Allison Colorado, Broad Communications A fellow in Broad's Gene Regulation Observatory, Bo Xia studies how the structure of the genome determines cell fate in both health and disease. In school, he loved imagining how molecules made up materials and whole organisms.

DNA 81

DNA Regulations Cell Biology Disease 81

Broad Institute

MARCH 27, 2025

A Novo Nordisk - Broad collaboration is connecting Denmark-based and Broad researchers By Corie Lok March 27, 2025 Breadcrumb Home A Novo Nordisk - Broad collaboration is connecting Denmark-based and Broad researchers The Novo Nordisk Foundation Center for Genomic Mechanisms of Disease has built a bridge between Danish and Broad scientists.

Research 75

Research DNA Regulations Disease 75

Codon

APRIL 24, 2025

We’re close to selling out of our second book —written in both text and DNA—and we recently launched a merchandise store. By analyzing how enrollment rates respond to changes in policy, funding, and disease targets, we can see which approaches most effectively accelerate pharmaceutical innovation. Cite: Jain, H.

Clinical Trials 122

Clinical Trials Trials Pharmaceutical Companies Licensing 122

Broad Institute

MAY 13, 2025

AI tool predicts potential drug targets by analyzing cell images By Corie Lok May 13, 2025 Breadcrumb Home AI tool predicts potential drug targets by analyzing cell images Researchers have developed a machine learning model that connects images of DNA structure to gene regulation.

DNA 52

DNA Drugs Regulations Molecular Biology 52

New Drug Approvals

APRIL 13, 2025

Retrieved 25 January 2025. “Recent advances in treating hypertriglyceridemia in patients at high risk of cardiovascular disease with apolipoprotein C-III inhibitors” Expert Opinion on Pharmacotherapy. January 2025. Archived from the original on 21 January 2025. Retrieved 21 January 2025. 1 October 2024.

FDA 62

FDA FDA Approval DNA Pharmaceuticals 62

New Drug Approvals

APRIL 5, 2025

9] In the body, treosulfan is converted into other compounds called epoxides which kill cells, especially cells that develop rapidly such as bone marrow cells, by attaching to their DNA while they are dividing. [9] TREOSULFAN C 6 H 14 O 8 S 2 MW 278.29 9] It belongs to the family of drugs called alkylating agents. [9] 11 October 2022.

FDA 62

FDA FDA Approval Production Treatment 62

KIF1A

NOVEMBER 1, 2024

2025 KAND Family & Scientific Engagement Conference We’re excited to announce the official dates for our 2025 KAND Conference! Mark your calendars for July 25-26, 2025, and plan to join us in Boston. Visit our 2025 KAND Conference webpage for all the details, links, and information as it is available. SAVE the DATE!

Treatment 52

Treatment Medical Schools Research Disease 52

LifeSciVC

MAY 28, 2025

But with the rise of Chinas innovative sector, the proliferation of biologic drugs coded by DNA, and more than dozen years of abundant capital, to name just a few things – the challenge has only gotten worse. As illustrated in LEKs May 2025 report Is Biopharma Doing Enough to Advance Novel Targets ?

Production 80

Production Marketing DNA Drugs 80

Codon

MARCH 19, 2025

Biosecurity is always a challenging question because our goal is to manipulate human biology to cure disease. White: The end goal is to have a platform that runs scientific intelligence at a scale that is good enough to work on all genes, all proteins, all diseases. Asimov Press (2025). We think about it a lot. Cite: Duoto, B.

Science 86

Science Research DNA Laboratories 86

Drug Target Review

MAY 8, 2025

Huntington’s disease (HD) is a devastating neurodegenerative disorder with profound generational impacts. This hereditary pattern makes HD particularly challenging from both medical and societal perspectives: a condition with a known genetic cause but, to date, no disease-modifying treatments.

Disease 111

Disease Treatment DNA Therapies 111

PLOS: DNA Science

FEBRUARY 27, 2025

Every year near the end of February, Rare Disease Day honors the 30 million people in the US who have 10,000+ rare medical conditions, and those who support them. Exciting interactions arise at rare disease conferences, at local, national, and international levels. To learn more and find ways to get involved, visit rarediseaseday.us.

Disease 74

Disease DNA FDA Therapies 74

Broad Institute

JANUARY 7, 2025

A clinical genomicist harnesses team-based science to help rare-disease patients By Allessandra DiCorato January 7, 2025 Breadcrumb Home A clinical genomicist harnesses team-based science to help rare-disease patients Heidi Rehm convinced labs and scientists to work together and share data.

Disease 121

Disease Science Laboratories Medical Schools 121

Broad Institute

JANUARY 16, 2025

By Allessandra DiCorato January 16, 2025 Credit: Scott Sassone, Broad Communications Tara McDonald (left) and Steve McCarroll (right) are coauthors of a new paper describing a biological mechanism underlying Huntington's disease. The cumulative death of many such cells leads to the symptoms of Huntingtons disease.

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Disease DNA Medical Schools RNA 130

PLOS: DNA Science

MAY 29, 2025

I love when fiction unfurls a compelling tale whose protagonist has an ultrarare genetic disease. I reviewed the book here at DNA Science. The Sirens Mimic Mermaids Middlesex is from 2002, The Covenant of Water from 2023, The Sirens , 2025. My most recent favorite is The Sirens.

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Disease DNA Science Government 79