RNA - Drug Discovery Digest

RNA that doesn't age

Science Daily: Pharmacology News

APRIL 5, 2024

Certain RNA molecules in the nerve cells in the brain last a life time without being renewed. RNAs are generally short-lived molecules that are constantly reconstructed to adjust to environmental conditions. Neuroscientists have now demonstrated that this.

RNA

RNA Disease Research

RNA editing: emerging from CRISPR’s shadow

BioPharma Drive: Drug Pricing

OCTOBER 22, 2024

Early study data from Wave Life Sciences suggests how editing RNA may yield viable medicines. Large and small drugmakers say such results are just the start.

RNA

RNA Science

Wave sees RNA editing validation in early trial results

BioPharma Drive: Drug Pricing

OCTOBER 16, 2024

The results provide the first clinical evidence of RNA editing, a burgeoning field that's drawn interest from biotechs and pharmaceutical companies alike.

RNA

RNA Pharmaceutical Companies Trials Pharmaceuticals

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Research shows how RNA 'junk' controls our genes

Science Daily: Pharmacology News

JULY 2, 2024

The new study focuses on critical snippets of RNA in the tiny, transparent roundworm Caenorhabditis elegans (C. The study provides a detailed map of the 3'UTR regions of RNA in C. 3'UTRs (untranslated regions) are segments of RNA involved in gene regulation.

RNA

RNA Research Regulations

Orna, a circular RNA specialist, acquires a buzzy startup

BioPharma Drive: Drug Pricing

MAY 23, 2024

After laying off staff late last year, Orna is expanding through the purchase of ReNAgade Therapeutics, which was built around technology designed to more effectively deliver RNA-based medicines.

RNA

Bayer partners with RNA drugmaker to develop new cancer therapies

BioPharma Drive: Drug Pricing

AUGUST 28, 2024

Under a collaboration with NextRNA Therapeutics, Bayer will access the biotech’s platform to target long, non-coding RNA interactions with small molecule drugs.

RNA

RNA Small Molecule Therapies Drugs

Researchers develop RNA-targeting technology for precisely manipulating parts of human genes

Science Daily: Pharmacology News

JUNE 24, 2024

Researchers have harnessed a bacterial immune defense system, known as CRISPR, to efficiently and precisely control the process of RNA splicing.

RNA

RNA Research Disease

Judo Bio debuts with $100M and a plan to take RNA drugs to the kidney

BioPharma Drive: Drug Pricing

OCTOBER 7, 2024

The Cambridge, Massachusetts-based biotech is using what it calls ligand-siRNA conjugates to reach drug targets in the kidney, which historically have been difficult to reach with RNA therapies.

RNA

RNA Therapies Drugs

New imaging method enables detailed RNA analysis of the whole brain

Science Daily: Pharmacology News

NOVEMBER 21, 2024

Researchers have developed a groundbreaking microscopy method that enables detailed three-dimensional (3D) RNA analysis at cellular resolution in whole intact mouse brains. The new method, called TRISCO, has the potential to transform our understanding of brain function, both in normal conditions and in disease, according to the new study.

RNA

RNA Disease Research

New lipid nanoparticle platform delivers mRNA to the brain through the blood-brain barrier

Science Daily: Pharmacology News

FEBRUARY 17, 2025

Scientists have developed a lipid nanoparticle system capable of delivering messenger RNA (mRNA) to the brain via intravenous injection, a challenge that has long been limited by the protective nature of the blood-brain barrier.

RNA

RNA Treatment Disease Drugs

Engineers design more powerful RNA vaccines

Science Daily: Pharmacology News

SEPTEMBER 7, 2023

By adding synergistic self-adjuvanting properties to Covid-19 RNA vaccines, researchers showed they could significantly boost the immune response generated in mice.

RNA

RNA Vaccine Engineering Immune Response

RNA for the first time recovered from an extinct species

Science Daily: Pharmacology News

SEPTEMBER 19, 2023

A new study shows the isolation and sequencing of more than a century-old RNA molecules from a Tasmanian tiger specimen preserved at room temperature in a museum collection. This resulted in the reconstruction of skin and skeletal muscle transcriptomes from an extinct species for the first time.

RNA

RNA Virus International Research

Roche partners with RNA editing biotech Ascidian

BioPharma Drive: Drug Pricing

JUNE 18, 2024

While the Swiss pharma recently pruned its neurology drug pipeline, it has shown interest in the possibilities of RNA-focused approaches like what Ascidian is exploring.

RNA

RNA Drugs

Biotech startup Airna raises $60M for RNA editing medicines

BioPharma Drive: Drug Pricing

JULY 31, 2024

Launched last September, the company is working on a treatment for alpha-1 antitrypsin deficiency, which it said could enter the clinic in 2025.

RNA

RNA Treatment

An ancient RNA-guided system could simplify delivery of gene editing therapies

Broad Institute

FEBRUARY 27, 2025

An ancient RNA-guided system could simplify delivery of gene editing therapies By Corie Lok February 27, 2025 Breadcrumb Home An ancient RNA-guided system could simplify delivery of gene editing therapies The programmable proteins are compact, modular, and can be directed to modify DNA in human cells.

RNA

RNA Therapies DNA Virus

Roche bets on Ionis RNA medicines with neuroscience deal

BioPharma Drive: Drug Pricing

SEPTEMBER 27, 2023

The Swiss drugmaker is restocking its pipeline by buying into preclinical Ionis medicines for Alzheimer’s and Huntington’s diseases.

RNA

RNA Disease

Wave readies clinical testing for first RNA editing therapy

BioPharma Drive: Drug Pricing

SEPTEMBER 5, 2023

The biotech, one of several advancing RNA editing medicines, expects to obtain “proof-of-mechanism” data in patients with the inherited disorder alpha-1 antitrypsin deficiency by next year.

RNA

RNA Therapies

Ipsen, Skyhawk to develop RNA-targeting pills for rare brain diseases

BioPharma Drive: Drug Pricing

APRIL 22, 2024

billion, is the latest research collaboration involving small molecule drugs that modify RNA. The deal, which could be worth up to $1.8

RNA

RNA Small Molecule Disease Research

Roche joins startup Remix in bet on another way to drug RNA

BioPharma Drive: Drug Pricing

JANUARY 3, 2024

The deal is the third Roche has formed with startups developing pills that target RNA, following similar pacts with Arrakis and Ribometrix.

RNA

RNA Drugs

Startup AIRNA emerges from stealth to bring RNA editing to a rare disease

BioPharma Drive: Drug Pricing

SEPTEMBER 19, 2023

AIRNA is one of several biotechs to debut plans for target alpha-1 antitrypsin deficiency in recent years, with competitors such as AlveoGene, Wave Life Sciences and Arrowhead Pharmaceuticals.

RNA

RNA Disease Science Pharmaceuticals

An RNAi renaissance is creating a new generation of startups

BioPharma Drive: Drug Pricing

NOVEMBER 6, 2024

Pioneers like Alnylam Pharmaceuticals have led the RNA interference field for years. Now, a crop of young biotechs is building on that foundation by taking the drugmaking technology in new directions.

RNA

RNA Pharmaceuticals

A covalent compound selectively inhibits RNA demethylase ALKBH5 rather than FTO

Covalent Modifiers

FEBRUARY 25, 2024

TD19 irreversibly modifies the residues C100 and C267, preventing ALKBH5 from binding to m6A-containing RNA. Moreover, TD19 displays good anticancer efficacy in acute myeloid leukemia and glioblastoma multiforme cell lines.

RNA

RNA Cell Based Assays Regulations Research

Life from a drop of rain: New research suggests rainwater helped form the first protocell walls

Science Daily: Pharmacology News

AUGUST 21, 2024

billion years ago, a critical step in the transition from tiny beads of RNA to every bacterium, plant, animal, and human that ever lived. New research shows that rainwater could have helped create a meshy wall around protocells 3.8

RNA

RNA Research

What gave the first molecules their stability?

Science Daily: Pharmacology News

AUGUST 2, 2024

A team has demonstrated a mechanism that could have enabled the first RNA molecules to stabilize in the primordial soup. When two RNA strands combine, their stability and lifespan increase significantly. How were complex molecules able to form and remain intact for prolonged periods without disintegrating?

RNA

FDA approves first-of-its-kind RNA drug for hemophilia

BioPharma Drive: Drug Pricing

MARCH 28, 2025

The Sanofi drug, known as fitusiran and now Qfitlia, was approved on Friday for a broad group of people with the rare bleeding disorder.

FDA Approval

FDA Approval RNA FDA Drugs

RNA editing specialist Korro to go public via reverse merger

BioPharma Drive: Drug Pricing

JULY 14, 2023

The deal with Frequency Therapeutics is the latest biotech reverse merger as private drugmakers still face challenges going public via an IPO.

RNA

Novartis buys a preclinical biotech and its RNA drug technology

BioPharma Drive: Drug Pricing

JULY 17, 2023

For $500 million, Novartis will acquire DTx Pharma and its preclinical neurological disease drugs, marking the Swiss company’s latest investment in gene-silencing medicines.

RNA

RNA Drugs Disease

Over 160,000 new viruses discovered by AI

Science Daily: Pharmacology News

OCTOBER 9, 2024

161,979 new RNA viruses have been discovered using a machine learning tool that researchers believe will vastly improve the mapping of life on Earth and could aid in the identification of many millions more viruses yet to be characterized.

Virus

Virus RNA Research

Genomic dark matter solves butterfly evolutionary riddle

Science Daily: Pharmacology News

AUGUST 30, 2024

The team discovered that an RNA molecule, rather than a protein as previously thought, plays a pivotal role in determining the distribution of black pigment on butterfly wings. Researchers have uncovered a surprising genetic mechanism that influences the vibrant and complex patterns on butterfly wings.

RNA

RNA Research

The critical role of comprehensive RNA sequencing in liquid biopsy for biomarker discovery and clinical trials

BioPharma Drive: Drug Pricing

JUNE 17, 2024

Elevate precision medicine: dose optimization and immune monitoring through advanced liquid biopsies.

Clinical Trials

Clinical Trials RNA Trials

Octopuses rewire their brains to adapt to seasonal temperature shifts

Science Daily: Pharmacology News

JUNE 8, 2023

The octopuses achieve this by editing their RNA, the messenger molecule between DNA and proteins. Researchers report that two-spot octopuses adapt to seasonal temperature shifts by producing different neural proteins under warm versus cool conditions.

RNA

RNA DNA Research

Life's building blocks in Asteroid Bennu samples

Science Daily: Pharmacology News

JANUARY 29, 2025

Scientists detected all five nucleobases -- building blocks of DNA and RNA -- in samples returned from asteroid Bennu by NASA's OSIRIS-REx mission.

RNA

RNA DNA

RNA editing biotech Airna adds $155M in funding

BioPharma Drive: Drug Pricing

APRIL 1, 2025

Airna will use the Series B round to launch a Phase 1/2 trial of its lead program for alpha-1 antitrypsin deficiency, a disease targeted by several other firms.

RNA

RNA Trials Disease

CDD Vault Update (February #2 2025): Advanced Macromolecules - Modified Bioconjugates Support in CDD Vault

Collaborative Drug

FEBRUARY 21, 2025

CDD Update February 2025 #2 New Macromolecule Mode Edit DNA, RNA & Peptides with Bioconjugate Support

RNA

RNA DNA

Exsilio debuts with $82M, pitching a ‘leap’ forward for genetic medicine

BioPharma Drive: Drug Pricing

JUNE 25, 2024

The company claims it can address some of the field’s limitations with medicines delivered via messenger RNA and lipid nanoparticles.

RNA

Editing without 'cutting': Molecular mechanisms of new gene-editing tool revealed

Science Daily: Pharmacology News

MAY 29, 2024

Functional analysis based on these structures also revealed how a 'prime editor' could achieve reverse transcription, synthesizing DNA from RNA, without 'cutting' both strands of the double helix. New research has determined the spatial structure of various processes of a novel gene-editing tool called 'prime editor.'

RNA

RNA DNA Therapies Treatment

Updated COVID shots from Pfizer, Moderna cleared by FDA

BioPharma Drive: Drug Pricing

AUGUST 22, 2024

The approval of both reformulated messenger RNA vaccines, which are designed to target the KP.2 2 virus strain, comes just ahead of the fall and winter seasons.

RNA

RNA FDA Virus Vaccine

Cryo-microscopy reveals nano-sized copy machine implicated in origin of life

Science Daily: Pharmacology News

JANUARY 16, 2024

RNA is thought to have sparked the origin of life by self-copying. Researchers have now revealed the atomic structure of an 'RNA copy machine' through cryo-EM. This breakthrough sheds light on a primordial RNA world and fuels advancements in RNA nanotechnology and medicine.

RNA

RNA Research

RNA: The most attractive target in recent viral diseases

Chemical Biology and Drug Design

DECEMBER 13, 2023

This up-to-date commentary/perspective article sheds light on the revolutionary approaches designed to target the principal viral weapon “RNA” used by RNA viruses (e.g., the severe acute respiratory syndrome coronavirus 2 “SARS-CoV-2”) to infect humans and spread infections, the genomic RNA strands.

RNA

RNA Disease Virus Drugs

Blood Test Spots Early Pancreatic Cancers With 97% Accuracy

Drugs.com

APRIL 8, 2024

MONDAY, April 8, 2024 -- A blood test appears capable of detecting early-stage pancreatic cancers with up to 97% accuracy, a new study reports.The test looks for eight small RNA particles and eight larger DNA markers shed by pancreatic cancers,

RNA

RNA DNA

RNA processing in health and disease: challenges and opportunities of the field

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

RNA Small Molecule Disease Assay Development

FDA denies expanded approval for Alnylam RNA drug

BioPharma Drive: Drug Pricing

OCTOBER 9, 2023

The agency rejected Alnylam’s application for approval of its medicine patisiran in people with a rare heart condition, setting back the company’s plans.

RNA

RNA FDA Drugs

Researchers develop 'in vivo' RNA-based gene editing model for blood disorders

Science Daily: Pharmacology News

JULY 27, 2023

In a step forward in the development of genetic medicines, researchers have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body.

RNA

RNA Research Therapies Disease

In Early Research, Heart Drugs Show Promise in Fighting Muscular Dystrophy

Drugs.com

JANUARY 3, 2024

3, 2024 -- A regularly prescribed class of heart medications might be capable of treating one of the most common forms of muscular dystrophy, a new study in mice suggests.Myotonic dystrophy type 1 (DM1) is caused by abnormal RNA. WEDNESDAY, Jan.

RNA

RNA Research Drugs

RNA that doesn't age

RNA editing: emerging from CRISPR’s shadow

Webinars

Trending Sources

Wave sees RNA editing validation in early trial results

Webinars

Research shows how RNA 'junk' controls our genes

Orna, a circular RNA specialist, acquires a buzzy startup

Bayer partners with RNA drugmaker to develop new cancer therapies

Researchers develop RNA-targeting technology for precisely manipulating parts of human genes

Judo Bio debuts with $100M and a plan to take RNA drugs to the kidney

New imaging method enables detailed RNA analysis of the whole brain

New lipid nanoparticle platform delivers mRNA to the brain through the blood-brain barrier

Engineers design more powerful RNA vaccines

RNA for the first time recovered from an extinct species

Roche partners with RNA editing biotech Ascidian

Biotech startup Airna raises $60M for RNA editing medicines

An ancient RNA-guided system could simplify delivery of gene editing therapies

Roche bets on Ionis RNA medicines with neuroscience deal

Wave readies clinical testing for first RNA editing therapy

Ipsen, Skyhawk to develop RNA-targeting pills for rare brain diseases

Roche joins startup Remix in bet on another way to drug RNA

Startup AIRNA emerges from stealth to bring RNA editing to a rare disease

An RNAi renaissance is creating a new generation of startups

A covalent compound selectively inhibits RNA demethylase ALKBH5 rather than FTO

Life from a drop of rain: New research suggests rainwater helped form the first protocell walls

What gave the first molecules their stability?

FDA approves first-of-its-kind RNA drug for hemophilia

RNA editing specialist Korro to go public via reverse merger

Novartis buys a preclinical biotech and its RNA drug technology

Over 160,000 new viruses discovered by AI

Genomic dark matter solves butterfly evolutionary riddle

The critical role of comprehensive RNA sequencing in liquid biopsy for biomarker discovery and clinical trials

Octopuses rewire their brains to adapt to seasonal temperature shifts

Life's building blocks in Asteroid Bennu samples

RNA editing biotech Airna adds $155M in funding

CDD Vault Update (February #2 2025): Advanced Macromolecules - Modified Bioconjugates Support in CDD Vault

Exsilio debuts with $82M, pitching a ‘leap’ forward for genetic medicine

Editing without 'cutting': Molecular mechanisms of new gene-editing tool revealed

Updated COVID shots from Pfizer, Moderna cleared by FDA

Cryo-microscopy reveals nano-sized copy machine implicated in origin of life

RNA: The most attractive target in recent viral diseases

Blood Test Spots Early Pancreatic Cancers With 97% Accuracy

RNA processing in health and disease: challenges and opportunities of the field

FDA denies expanded approval for Alnylam RNA drug

Researchers develop 'in vivo' RNA-based gene editing model for blood disorders

In Early Research, Heart Drugs Show Promise in Fighting Muscular Dystrophy

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