RNA - Clinical Research Informer

RNA editing: emerging from CRISPR’s shadow

Bio Pharma Dive

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 Life Science Medicine

Wave sees RNA editing validation in early trial results

Bio Pharma Dive

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 Trials

Moderna founder's next big play in RNA raises $440 million

Bio Pharma Dive

AUGUST 30, 2021

Just a few months out of stealth mode, Laronde and its "endless RNA" technology have caught the attention of well-known investors like Fidelity, T. Rowe Price Associates and Invus.

RNA

Orna, a circular RNA specialist, acquires a buzzy startup

Bio Pharma Dive

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

RNA Medicine

Moderna founder launches new biotech, looking to cure diseases through 'endless RNA'

Bio Pharma Dive

MAY 10, 2021

Laronde comes equipped with $50 million in funding from Flagship Pioneering and a goal to upend how diseases are treated with long-lasting RNA medicines.

RNA

RNA Medicine

Moderna founder unveils new drug company focused on a different kind of RNA

Bio Pharma Dive

NOVEMBER 9, 2021

The biotech is researching how to make medicines from transfer RNA molecules. Encouraged by Moderna's success, Flagship Pioneering has been busy creating startups like Alltrna, which launched Tuesday.

RNA

RNA Medicine Drugs Research

Bayer partners with RNA drugmaker to develop new cancer therapies

Bio Pharma Dive

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 Development Drugs

Next-generation RNA technologies: making longer-lasting drugs with a broader reach

Bio Pharma Dive

AUGUST 23, 2022

At least 10 biotechnology startups are trying to use RNA molecules in new ways to make medicines. Here’s a look at where they stand.

RNA

RNA Medicine Drugs

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

Bio Pharma Dive

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 Drugs

Drugging RNA with pills: small molecules for a big frontier

Bio Pharma Dive

SEPTEMBER 6, 2022

A handful of startups have formed in recent years with plans to target RNA with small molecules. Many have caught the attention of big drugmakers.

RNA

RNA Drugs

As Arrowhead falters, lungs remain a tough target for RNA drugs

Bio Pharma Dive

JULY 6, 2021

The biotech's decision to pause testing of a cystic fibrosis drug is the latest reminder of the difficulty of delivering RNA medicines into the lungs.

RNA

RNA Drugs Medicine

Amgen partners with Arrakis to develop drugs aimed at RNA

Bio Pharma Dive

JANUARY 11, 2022

Arrakis, a specialist in designing oral drugs that target RNA, will receive $75 million under the deal, which could be worth "several billion dollars" more.

RNA

RNA Drugs Development

Gatehouse Bio and AstraZeneca to develop RNA therapeutics for heart failure

Pharmaceutical Technology

SEPTEMBER 9, 2022

US-based biotech company Gatehouse Bio is extending its collaboration with AstraZeneca for developing RNA therapeutics to treat heart failure with preserved ejection fraction (HFpEF). Gatehouse Bio is engaged in developing small RNA-based therapeutics using AI technology.

RNA

RNA Development Gene Therapy Gene

Ascidian starts up with $50M and a twist on RNA editing

Bio Pharma Dive

OCTOBER 12, 2022

The startup, run by Translate Bio cofounder Romesh Subramanian, believes its RNA “exon editing” approach could have long-lasting effects without the risks that come with editing DNA.

RNA

RNA DNA

China-based biotech licenses Sanofi's RNA drug technology

Bio Pharma Dive

JULY 25, 2022

The Lilly-backed Rona Therapeutics gains rights to a platform for small interfering RNA, along with a slate of preclinical candidates aimed at targets in the liver and other tissues.

RNA

RNA Licensing Licensing Drugs

Venture capital pours more money into RNA medicines with the launch of Replicate

Bio Pharma Dive

SEPTEMBER 8, 2021

The company, which focuses on self-replicating RNA, comes equipped with $40 million from Apple Tree Partners and a stamp of approval from the former research head at Biogen.

RNA

RNA Medicine Research

Seattle institute lands $18m funding to develop RNA vaccine for chikungunya

Pharmaceutical Technology

AUGUST 3, 2023

AAHI is developing a novel RNA vaccine that overcomes manufacturing and storage limitations to increase accessibility.

RNA

RNA Vaccination Vaccine Development

Beam-linked startup launches with an eye on the next generation of RNA drugs

Bio Pharma Dive

SEPTEMBER 7, 2022

“We see a huge opportunity in the setting of RNA-based therapies to overcome some of the shortcomings that have been there with the first generation of RNA companies,” said former Alnylam CEO and Orbital Therapeutics co-founder John Maraganore.

RNA

RNA Drugs

New UK intracellular drug delivery centre to support RNA development

Pharmaceutical Technology

JUNE 1, 2023

A new intracellular drug delivery centre will be established in the UK to support potential ribonucleic acid (RNA) vaccines and therapeutics , as well as the development of innovative drug delivery technologies. It will focus on studying and developing new lipid nanoparticle (LNP) formulations for the delivery of RNA medicine.

Drug Delivery

Drug Delivery RNA Development Drugs

Roche partners with RNA editing biotech Ascidian

Bio Pharma Dive

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

BioNTech’s RNA vaccine sparks potential in pancreatic cancer

Pharmaceutical Technology

MAY 11, 2023

Results published in Nature for a personalised pancreatic cancer vaccine that uses neoantigens from patients’ tumours have lent further support to early positive signals. The vaccine, developed by BioNTech, led to half of the patients with pancreatic cancer in the Phase I trial remaining cancer-free 18 months later.

RNA

RNA Vaccination Vaccine Trials

Triastek and BioNTech to develop 3D-printed RNA therapeutics

Pharmaceutical Technology

JULY 24, 2024

BioNTech and Triastek have entered a research partnership focusing on the development of 3D-printed oral RNA therapeutics.

RNA

RNA Development Research

J&J to explore RNA reprogramming with Remix research deal

Bio Pharma Dive

FEBRUARY 17, 2022

The agreement with the Cambridge-based startup is the latest to reflect growing large pharma company interest in targeting RNA with small molecules.

RNA

RNA Pharma Companies Research

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

Bio Pharma Dive

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 Development

Biotech startup Airna raises $60M for RNA editing medicines

Bio Pharma Dive

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 Medicine

Wave readies clinical testing for first RNA editing therapy

Bio Pharma Dive

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 Medicine

Novo to acquire Dicerna for more than $3B amid RNA drug resurgence

Bio Pharma Dive

NOVEMBER 18, 2021

The acquisition is a rare deal for Novo Nordisk, which has been partnered with Dicerna since 2019.

RNA

RNA Drugs

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

Bio Pharma Dive

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 Development Drugs Research

ReNAgade debuts with $300M and an ‘all-in-one’ system for delivering RNA drugs

Bio Pharma Dive

MAY 23, 2023

Staffed by many former Alnylam and Moderna employees, the startup is developing technologies that could be useful in a variety of RNA drugmaking methods.

RNA

RNA Drugs Development

Roche adds to RNA drug deal streak with Ribometrix partnership

Bio Pharma Dive

JANUARY 6, 2021

The companies' pact is the latest addition to a growing list of deals involving startups developing chemical drugs to target RNA molecules.

RNA

RNA Drugs Development

AstraZeneca gives up on Ionis’ RNA drug for heart disease

Bio Pharma Dive

SEPTEMBER 23, 2022

The British drugmaker decided the medicine, which works similarly to Novartis’s Leqvio, wasn’t potent enough to justify further testing.

RNA

RNA Medicine Drugs

CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Pharmaceutical Technology

JULY 29, 2022

Last week, CAMP4 Therapeutics announced the close of a $100 million Series B round , which will be used to advance their regulatory RNA (regRNA)-focused programs. According to Bumcrot, regRNAs are “RNAs that arise out of the non-coding genome”. Their platform identifies which regRNAs control the specific gene of interest.

RNA

RNA Gene Expression Gene Gene Therapy

Self-amplifying RNA and vaccine innovation: getting from concept to clinic

Pharmaceutical Technology

MAY 28, 2024

Biomanufacturers using self-amplifying RNA, or saRNA, could enjoy significant advantages

RNA

RNA Vaccination Vaccine

Roche bets on Ionis RNA medicines with neuroscience deal

Bio Pharma Dive

SEPTEMBER 27, 2023

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

Medicine

Medicine RNA

AstraZeneca strikes deal with university spinoff to turn RNA into drugs

Bio Pharma Dive

SEPTEMBER 23, 2021

The British drugmaker is investing in VaxEquity, a biotech founded last year to explore a twist on the mRNA technology that Moderna and BioNTech have made famous.

RNA

RNA Drugs

ReNAgade Therapeutics enters RNA therapy arena with $300m

Pharmaceutical Technology

MAY 23, 2023

ReNAgade Therapeutics has burst onto the RNA technology scene by announcing a $300m financing round. With its delivery system, Cambridge, Massachusetts-based biotech says it aims to “address major limitations in RNA therapeutics by enabling the delivery of RNA medicines to previously inaccessible tissues and cells in the body.”

RNA

RNA Gene Editing Gene Vaccine

Flamingo gets grant from VLAIO to accelerate RNA-focused oncology portfolio

Pharmaceutical Technology

JUNE 16, 2023

from Flanders Innovation & Entrepreneurship (VLAIO) to further advance its oncology portfolio targeting RNA. It will also support the firm’s preclinical effort on the long non-coding RNA (LncRNA) programme, FTX-001, that targets MALAT-1. Flamingo Therapeutics has received a research grant of €1.7m

RNA

RNA Gene Therapy Gene Clinical Trials

An RNAi renaissance is creating a new generation of startups

Bio Pharma Dive

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

Merck pays startup Orna $150M as ‘circular RNA’ attracts industry interest

Bio Pharma Dive

AUGUST 16, 2022

The pharmaceutical company has formed an alliance with Orna and invested $100 million in its Series B round, hoping the young biotech’s approach could lead to multiple new vaccines and drugs.

RNA

RNA Vaccination Vaccine Drugs

Lilly joins RNA editing race with ProQR deal

Bio Pharma Dive

SEPTEMBER 9, 2021

An agreement with the Dutch biotech, which could be worth as much as $1.3 billion, expands Lilly's work in genetic medicine research.

RNA

RNA Genetics Medicine Research

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

Bio Pharma Dive

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 Life Science

Beyond Covid-19: the disruptive potential of RNA-based therapeutics

Pharmaceutical Technology

MARCH 1, 2023

Further, the acceptance of new mRNA vaccines has rejuvenated activity within previously established categories of RNA therapeutics including lifesaving antisense technologies. These include antisense oligonucleotides (ASO), RNA interference (RNAi), and RNA aptamers.

RNA

RNA Vaccine Vaccination Genetics

Moderna, Merck data support claim of cancer vaccine’s promise

Bio Pharma Dive

APRIL 16, 2023

Data presented at AACR suggest a personalized shot made with messenger RNA may amplify the effects of a widely prescribed cancer immunotherapy.

RNA

Pfizer begins plan to challenge Shingrix with an mRNA vaccine

Bio Pharma Dive

FEBRUARY 10, 2023

The pharma and partner BioNTech have kicked off the first human trial of a messenger RNA shot for shingles, believing there’s room to clear the high bar set by GSK’s rival vaccine.

Vaccination

Vaccination Vaccine RNA Trials

RNA editing: emerging from CRISPR’s shadow

Wave sees RNA editing validation in early trial results

Trending Sources

Moderna founder's next big play in RNA raises $440 million

Orna, a circular RNA specialist, acquires a buzzy startup

Moderna founder launches new biotech, looking to cure diseases through 'endless RNA'

Moderna founder unveils new drug company focused on a different kind of RNA

Bayer partners with RNA drugmaker to develop new cancer therapies

Next-generation RNA technologies: making longer-lasting drugs with a broader reach

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

Drugging RNA with pills: small molecules for a big frontier

As Arrowhead falters, lungs remain a tough target for RNA drugs

Amgen partners with Arrakis to develop drugs aimed at RNA

Gatehouse Bio and AstraZeneca to develop RNA therapeutics for heart failure

Ascidian starts up with $50M and a twist on RNA editing

China-based biotech licenses Sanofi's RNA drug technology

Venture capital pours more money into RNA medicines with the launch of Replicate

Seattle institute lands $18m funding to develop RNA vaccine for chikungunya

Beam-linked startup launches with an eye on the next generation of RNA drugs

New UK intracellular drug delivery centre to support RNA development

Roche partners with RNA editing biotech Ascidian

BioNTech’s RNA vaccine sparks potential in pancreatic cancer

Triastek and BioNTech to develop 3D-printed RNA therapeutics

J&J to explore RNA reprogramming with Remix research deal

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

Biotech startup Airna raises $60M for RNA editing medicines

Wave readies clinical testing for first RNA editing therapy

Novo to acquire Dicerna for more than $3B amid RNA drug resurgence

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

ReNAgade debuts with $300M and an ‘all-in-one’ system for delivering RNA drugs

Roche adds to RNA drug deal streak with Ribometrix partnership

AstraZeneca gives up on Ionis’ RNA drug for heart disease

CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Self-amplifying RNA and vaccine innovation: getting from concept to clinic

Roche bets on Ionis RNA medicines with neuroscience deal

AstraZeneca strikes deal with university spinoff to turn RNA into drugs

ReNAgade Therapeutics enters RNA therapy arena with $300m

Flamingo gets grant from VLAIO to accelerate RNA-focused oncology portfolio

An RNAi renaissance is creating a new generation of startups

Merck pays startup Orna $150M as ‘circular RNA’ attracts industry interest

Lilly joins RNA editing race with ProQR deal

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

Beyond Covid-19: the disruptive potential of RNA-based therapeutics

Moderna, Merck data support claim of cancer vaccine’s promise

Pfizer begins plan to challenge Shingrix with an mRNA vaccine

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