The Pharma Data

JUNE 6, 2025

Sanofi Highlights Robust Pipeline of Innovations in Rare Blood Disorders at ISTH 2025 Sanofi is poised to take center stage at the 33rd Congress of the International Society on Thrombosis and Haemostasis (ISTH), set to be held in Washington, D.C., from June 21 to 25, 2025. and EU, in late 2024 as the first therapy of its kind.

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Disease Therapies Treatment International 40

Alta Sciences

FEBRUARY 19, 2025

Regulatory Guidance for Oligonucleotide Bioanalysis in Drug Development pmjackson Wed, 02/19/2025 - 21:30 The unique physicochemical properties of oligonucleotides require the use of specialized bioanalytical approaches, with key considerations including selectivity and specificity, sensitivity, stability, and matrix effects.

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Drug Development Clinical Pharmacology Immune Response Drugs 52

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). Academic specializing in RNA editing).

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RNA Protein Expression DNA Science 67

Drug Target Review

DECEMBER 16, 2024

Since their first approvals in 2017, autologous CAR T-cell therapies have revolutionised the treatment of blood cancers, offering hope where previous options had failed. Consequently, these therapies come with significant burdens and are often underutilised despite their transformative patient outcomes.

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The Pharma Data

JUNE 9, 2025

Alnylam Secures European Commission Approval for AMVUTTRA® to Treat Cardiomyopathy in ATTR Amyloidosis Alnylam Pharmaceuticals , a global leader in RNA interference (RNAi) therapeutics, has received European Commission (EC) approval for an expanded indication of its RNAi drug AMVUTTRA® (vutrisiran).

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Treatment Disease RNA Therapies 40

Codon

JUNE 5, 2025

The treatment, now known as Casgevy, became the first CRISPR-based therapy to gain FDA approval, in 2023. The core of the CRISPR immune response is a guide RNA (gRNA) that binds to a CRISPR-associated (Cas) protein. Collectively, these repeat-protospacer regions are known as CRISPR arrays. Visit our website to get a copy.

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DNA RNA Clinical Trials Engineering 83

Broad Institute

FEBRUARY 26, 2025

Why brain cancer is often resistant to immunotherapy By Allessandra DiCorato February 26, 2025 Breadcrumb Home Why brain cancer is often resistant to immunotherapy Researchers find four coordinated gene expression programs in immune cells from glioma tumors, including two that could lead to immunotherapy resistance. Online February 26, 2025.

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BioPharma Drive: Drug Pricing

JUNE 24, 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.

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RNA Drugs Therapies Licensing 219

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] ).

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Drug Development Drugs Clinical Trials Therapies 56

Codon

APRIL 14, 2025

Some, like predicting microbial growth rate from genetics , predicting CRISPR editing efficiency at a locus given a guide RNA , and protein stability prediction are in progress but would benefit from more support, especially to make solutions widely applicable and available to potential users. ” Asimov Press (2025).

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Protein Expression Engineering Research RNA 94

BioPharma Drive: Drug Pricing

JULY 16, 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. Some Wall Street analysts predicted sales might top $2 billion in 2025. You can unsubscribe at anytime.

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Therapies FDA Clinical Trials Licensing 301

BioPharma Drive: Drug Pricing

JULY 10, 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. Kennedy Jr., who has pushed for policy changes around mRNA vaccines in particular.

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Vaccine FDA Therapies Licensing 231

Broad Institute

JUNE 25, 2025

By Allessandra DiCorato June 25, 2025 Credit: Xavier and Smillie labs Microscopy image shows intestinal tissue from a Crohn’s patient with fibrosis. Fibrosis affects every organ in the body, but generally the therapies that are available to correct or reverse fibrosis work poorly,” Xavier said.

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Disease Medical Schools RNA Molecular Biology 111

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

MARCH 14, 2025

Advancing the Future of Biomarker-Driven Immunotherapy swheeler Fri, 03/14/2025 - 13:43 Mon, 04/21/2025 - 12:00 Resource Type Webinar Eduard Porta-Pardo Can spatial biomarkers revolutionize patient stratification for immune checkpoint inhibitors?

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RNA Clinical Trials Therapies Trials 59

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.

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Engineering DNA RNA Therapies 59

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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Small Molecule Trials Therapies Disease 143

Drug Target Review

JULY 1, 2024

Using the 10X Genomics Chromium platform, we conducted ribonucleic acid (RNA) sequencing on the midbrain’s PAG region in animals treated with SRP-001, ApAP and a vehicle control, focusing on gene expression changes related to pain processing. was founded in 2016 to develop safer, non-opioid therapies for acute and chronic pain.

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Treatment Clinical Trials Therapies Molecular Biology 59

Drug Target Review

NOVEMBER 29, 2023

Our primary focus is to design and develop RNA therapies for liver diseases. Our team of chemists then uses this information to develop novel therapies. By doing this, we get a superior understanding of which therapies will and will not work in human biology before a clinical trial.

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Therapies Disease RNA Engineering 102

Codon

SEPTEMBER 29, 2024

But, regardless of which was first, they all operated with the same core data as their mechanism for understanding life: messenger RNA ( mRNA ). It suggested that, in the future, certain classes of drugs, specifically genetic therapies, could be screened entirely virtually via models of life. As such, they were quietly abandoned.

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Engineering Virus DNA Therapies 145

Codon

MAY 15, 2023

Made from a Cas9 nickase (the histidine at amino acid position 840 is swapped for an alanine) fused to a reverse transcriptase enzyme (which makes DNA from RNA; transcription in reverse), prime editing clinical trials are expected to begin by 2025. Gene Therapy. Molecular Cell. Nature Biotechnology. Cell Systems. Cell Systems.

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DNA Bioinformatics Engineering Virus 52

Codon

MAY 15, 2023

Made from a Cas9 nickase (the histidine at amino acid position 840 is swapped for an alanine) fused to a reverse transcriptase enzyme (which makes DNA from RNA; transcription in reverse), prime editing clinical trials are expected to begin by 2025. Gene Therapy. Molecular Cell. Nature Biotechnology. Cell Systems. Cell Systems.

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DNA Bioinformatics Engineering Virus 52

BioPharma Drive: Drug Pricing

JUNE 30, 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. as early as the 2026-2027 flu season. Sign up A valid email address is required.

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Vaccine Marketing Trials Clinical Trials 100

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.

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DNA Laboratories RNA Disease 133

Elrig

MARCH 10, 2025

Research & Innovation 2025 Keynote Speakers Announced Cambridge, UK, 24 January 2025: The European Laboratory Research & Innovation Group (ELRIG), a not-for-profit, volunteer-led organisation for the drug discovery community, today announced the keynote speakers for Research & Innovation 2025.

Research 52

Research Laboratories Medical Schools Engineering 52

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.

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DNA RNA Science Research 131

Drug Target Review

APRIL 1, 2025

Advances in biotechnology are driving significant progress in the treatment of rare diseases, making it possible to develop targeted therapies for previously untreatable conditions. Founded in 2021, SynaptixBio is working to develop therapies for TUBB4A leukodystrophies – a group of rare and severe neurological disorders.

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Disease Therapies Clinical Trials Cell Biology 52

keypoint

JANUARY 8, 2025

Our first Fellows Spotlight of 2025 goes to Keystone Symposia Fellow Dr. Abigael Cheruiyot! Studying RNA surveillance mechanisms, they identified nonsense mediated decay as a potential target for novel cancer therapies. She is very passionate about both science and promoting equity within the research community.

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Medical Schools RNA Laboratories Doctors 59

Codon

FEBRUARY 19, 2025

ESM is a protein language model that captures certain aspects of protein structure and function, for example, but is blind to DNA or RNA-level features that drive cell behavior. Evo 2 was trained on DNA sequences but can make predictions about myriad aspects of biology, including RNA stability and protein structures. & McCarty N.

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DNA Laboratories Engineering RNA 109

Drug Target Review

NOVEMBER 27, 2024

For example, the European Medicines Agency (EMA), in its Regulatory Science Strategy to 2025 , has highlighted the critical role of biomarker discovery, qualification and utilisation in accelerating precision medicine.

Drug Development 52

Drug Development Regulations Laboratories FDA 52

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.

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Disease Treatment DNA Therapies 111

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.

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Therapies RNA Packaging Virus 56

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. Online January 16, 2025. Funding This work was supported by CHDI Foundation, Inc., DOI: 10.1016/j.cell.2024.11.038.

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Disease DNA Medical Schools RNA 130

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.

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Medical Schools Treatment Bioinformatics Hospitals 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 deadly structure allows it to kill its victims by preventing their cells from making messenger RNA (mRNA) through inhibition of the enzyme RNA Polymerase II. million people worldwide.

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Drugs DNA RNA Laboratories 109

Codon

APRIL 28, 2025

Each cell contains messenger RNA (mRNA) that carries instructions for making proteins, so Prasher first extracted mRNA from Aequorea and converted it into complementary DNA (cDNA). ” Asimov Press (2025). Prasher set out to find the GFP gene, and then clone it, by constructing a gene library from Aequorea jellyfish tissue.

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DNA Engineering Research Virus 81

Codon

FEBRUARY 5, 2025

The researchers also ran follow-up experiments in the laboratory; cell lines carrying the same mutations as those found in the monkey had similar RNA expression patterns as cells taken from Parkinson’s patients. ” Asimov Press (2025). If you’re interested, please contact him at trevor[at]highwaypharm.com.

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Disease Treatment Research RNA 52