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. wasn’t the first patient to receive base editing therapy. Some of these treatments, like K.J.’s,

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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. Theres still a long way to go to make this a therapy, Minikel said. Online January 14, 2025. Nature Medicine.

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Disease DNA Protein Production Treatment 144

New Drug Approvals

MAY 17, 2025

Retrieved 17 April 2025. ^ “Qalsody- tofersen injection” DailyMed. Jump up to: a b New Drug Therapy Approvals 2023 (PDF). It is administered as an intrathecal injection. [3] 3] Tofersen was approved for medical use in the United States in April 2023, [3] [6] and in the European Union in May 2024. [4] 3 November 2006.

FDA Approval 62

FDA Approval FDA Treatment Clinical Trials 62

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

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

Codon

JUNE 5, 2025

The treatment, now known as Casgevy, became the first CRISPR-based therapy to gain FDA approval, in 2023. Certain CRISPR components can add short DNA sequences from the genomes of defeated viruses into the bacterium’s own genome, creating a type of protective “memory.” pyogenes , making it more specific.

DNA 83

DNA RNA Clinical Trials Engineering 83

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. The torso was treated with a partial programming gene therapy.

Engineering 68

Engineering Therapies DNA Trials 68

BioPharma Drive: Drug Pricing

JUNE 11, 2025

Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. Among those who had gotten another such therapy, the rates were 52% and 62% in those trials.

FDA 270

FDA Drugs Therapies Science 270

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. These results are notable given the poor response of NRG1 fusion driven cancers to chemo- and immuno-therapies. months (see [link] ).

Drug Development 56

Drug Development Drugs Clinical Trials Therapies 56

Codon

APRIL 14, 2025

DNA sequencing and synthesis costs have dropped precipitously, in part, because maintaining Carlson curves became a rallying point for the field. Stephen Malina is Head of Engineering at Dyno Therapeutics, a company using machine learning to design better viral vectors for gene therapy. ” Asimov Press (2025).

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Protein Expression Engineering Research RNA 94

Fierce BioTech

FEBRUARY 14, 2025

Nonviral CRISPR-based knock-in of chimeric antigen receptor transgenes into T cells for cell therapy dwunderlin Fri, 02/14/2025 - 14:52 Thu, 04/03/2025 - 11:00 Resource Type Webinar Krishanu Saha, PhD Max Sellman Join us for an insightful webinar on the latest advancements in cell and gene therapy, focusing on the innovative Cas9-CLIPT technology.

Therapies 52

Therapies DNA Production 52

Drug Target Review

JANUARY 8, 2024

Contrary to popular belief, ageing is not caused by just random wear and tear of our bodies over time but is instead caused by a discrete set of biological mechanisms that we now better understand and can target with therapies. a clinical-stage private biopharma company developing therapies for neurological and psychiatric diseases.

Science 145

Science Disease FDA Approval Pharmaceutical Companies 145

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

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. In most cancers, the tumour evolves by acquiring mutations that confer growth advantages or resistance to therapies. This allows the cancer to evolve and develop resistance to the therapy.

Bioinformatics 105

Bioinformatics Immune Response DNA Vaccine 105

Codon

SEPTEMBER 29, 2024

mRNA was the intermediate stage between DNA and protein, a dynamic entity that shifted depending on the second-to-second needs of the cell, able to point out if a cell was cancerous or stressed, what kind of cell it was, and so on. The 2025 genetic network paper fit that bill exactly. As such, they were quietly abandoned.

Engineering 145

Engineering Virus DNA Therapies 145

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

BioPharma Drive: Drug Pricing

JUNE 9, 2025

Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. You can unsubscribe at anytime. The implications of this shift are profound.

Clinical Trials 141

Clinical Trials Drugs Therapies Clinical Supply 141

BioPharma Drive: Drug Pricing

JUNE 5, 2025

Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. By Delilah Alvarado • May 13, 2025 Keep up with the story. You can unsubscribe at anytime.

FDA 130

FDA Therapies Clinical Supply Licensing 130

BioPharma Drive: Drug Pricing

JUNE 9, 2025

Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. 3 Digital Behavioral Therapy for Fibromyalgia Ep. You can unsubscribe at anytime. By Jonathan Gardner • Sept.

Trials 130

Trials Clinical Supply Clinical Trials Therapies 130

BioPharma Drive: Drug Pricing

JUNE 12, 2025

Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. By Jacob Bell • May 6, 2025 Keep up with the story. You can unsubscribe at anytime.

Small Molecule 130

Small Molecule Packaging Therapies Pharmacy 130

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

Fierce BioTech

APRIL 23, 2025

Overcoming Critical DNA Synthesis Challenges in Viral Vector-Mediated Gene Therapy Development Overcome gene synthesis roadblocks. Discover how enzymatic DNA synthesis enables production of complex, high-GC, and regulatory sequencesunlocking progress where traditional methods fall short. Download now! Download now!

DNA 52

DNA Therapies Production Research 52

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

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

The same model can also generate brand-new DNA sequences at the scale of yeast chromosomes or small bacterial genomes. AI Architectures Before explaining how Evo 2 could make bioengineering more predictable, it helps to first understand how AI models actually “learn” the language of DNA.

DNA 109

DNA Laboratories Engineering RNA 109

New Drug Approvals

APRIL 13, 2025

Retrieved 25 January 2025. New Drug Therapy Approvals 2024 (PDF). January 2025. Archived from the original on 21 January 2025. Retrieved 21 January 2025. Retrieved 22 February 2025. ^ “Tryngolza (olezarsen) approved in U.S. 1] [2] It is given by injection under the skin. [1] 19 December 2024. -D-GALACTOPYRANOSYLOXY)HEXYLAMINO)-3-OXOPROPOXYMETHYL))METHYL)AMINO-5-OXOPENTANAMIDO)HEXYL))PHOSPHO)-2′-O-(2-METHOXYETHYL)-P-THIOADENYLYL-(3′-O-&

FDA 62

FDA FDA Approval DNA Pharmaceuticals 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 76

Production Marketing DNA Drugs 76

Codon

JANUARY 2, 2025

Embracing the book’s technology theme, we did something very special: With the help of three companies — CATALOG, Imagene, and Plasmidsaurus — we’ve encoded a complete copy of the book into DNA, thus merging bits with atoms. We’ve made 1,000 DNA capsules in total.

DNA 133

DNA Laboratories Packaging Vaccine 133

PLOS: DNA Science

FEBRUARY 27, 2025

” I’ve attended Rare Disease Day celebrations, and befriended many families while and since writing The Forever Fix: Gene Therapy and the Boy Who Saved It , published in 2012. DNA Science has covered Rare Disease Day for years – links are at the end of this post. Every year, February 28 is “A Day to be Heard.”

Disease 71

Disease DNA FDA Therapies 71

Drug Target Review

MAY 8, 2025

This combination of single-dose treatment with allele selectivity represents a unique approach among investigational HD therapies in development. This fundamentally different approach combining the durability of a single-dose therapy with the precision of allele-selective editing addresses a critical unmet need in HD treatment.

Disease 111

Disease Treatment DNA Therapies 111

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 researchers found that DNA tracts with 40 or more CAG repeats grow until they are hundreds of repeats long.

Disease 130

Disease DNA Medical Schools RNA 130

SugarCone Biotech

APRIL 8, 2025

Astra Zenecas purchase of Esobiotec with a $425M upfront has rekindled interest in CAR-T cell therapy technologies, and in particular, the in vivo cell therapy companies. In vivo CAR-T cell therapy requires that genetic material be delivered to the desired immune cells while those cells are still inside the patient.

Therapies 56

Therapies RNA Packaging Virus 56

Codon

APRIL 28, 2025

This was before DNA clones could produce these proteins synthetically, and few researchers were willing to endure the grueling work of collecting and cutting jellyfish. Prasher used those sequences to design short, synthetic DNA probes that matched what he believed the corresponding DNA would look like.

DNA 81

DNA Engineering Research Virus 81

Broad Institute

MAY 20, 2025

Scientists build cell line atlas of deadly cancers of the bile ducts and gallbladder By Leah Eisenstadt May 20, 2025 Breadcrumb Home Scientists build cell line atlas of deadly cancers of the bile ducts and gallbladder Researchers reveal new subtypes and potential therapeutic targets for biliary tract cancers, which lack effective treatments.

Medical Schools 133

Medical Schools Treatment Bioinformatics Research 133

Codon

MAY 29, 2025

These abilities are precisely what make them promising as potential therapies, particularly as we find ways to enhance their safety profiles. This is the step after the initial transcription of DNA into mRNA by RNA Polymerase II, the protein to which alpha amanitin binds. In both cases, peptide and protein production stops.

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Drugs DNA RNA Laboratories 109