Drugs and RNA - Clinical Research Informer

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

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

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

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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

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

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

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

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

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

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

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

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

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

Bio Pharma Dive

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 Drugs

Novartis buys a preclinical biotech and its RNA drug technology

Bio Pharma Dive

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.

Gene Silencing

Gene Silencing RNA Drugs Gene

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

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

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

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. In preparation for this, drug manufacturing will begin later this year and will be outsourced to external contract manufacturing organisations (CMOs).

RNA

RNA Gene Expression Gene Gene Therapy

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

CSL says drug for rare swelling disorder succeeds in late-stage study

Bio Pharma Dive

AUGUST 18, 2022

The company plans to soon seek approval of a once-monthly preventive drug for hereditary angioedema, a disease other drugmakers are targeting with gene editing and RNA medicines.

Gene Editing

Gene Editing RNA Drugs Gene

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

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

A first-of-its-kind mRNA drug falls short in cystic fibrosis

Bio Pharma Dive

MARCH 18, 2021

A treatment that sprays synthetic messenger RNA into the lungs failed to help cystic fibrosis patients, a blow to efforts to use the technology for more than vaccines.

RNA

RNA Vaccination Vaccine Drugs

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

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

KCL study reveals particles in cows’ milk effective for oral delivery of RNA drugs

Pharma Times

APRIL 29, 2024

The method has the potential to be used to deliver RNA to the bloodstream for a range of diseases

RNA

RNA Drugs

DCGI adds IVD devices for diagnosis of Covid-19, RNA & DNA extraction kits in Class C risk category under MDR-2017

AuroBlog - Aurous Healthcare Clinical Trials blog

NOVEMBER 1, 2023

The Drug Controller General of India (DCGI) has added in-vitro diagnostic (IVD) medical devices including those for diagnosis of Covid-19, ribonucleic acid (RNA) and deoxyribonucleic acid (DNA) extraction kits, among others into the Class C risk category under the Medical Devices Rules (MDR), 2017.

RNA

RNA DNA In-Vitro Drugs

Tetra and Cellvera partner to develop oral Covid-19 treatment

Pharmaceutical Technology

SEPTEMBER 8, 2022

A first-in-human drug product, ARDS-003 contains the active pharmaceutical agent, Onternabez. It is claimed to have outperformed an antiviral drug in lowering various proinflammatory mediators linked to hyperinflammation and dysfunction of the immune system after viral infection.

Development

Development RNA Containment Drugs

Daiichi Sankyo and Depixus partner to accelerate RNA-targeted drug discovery

Pharmaceutical Technology

DECEMBER 13, 2023

Daiichi Sankyo plans to leverage the French startup’s technology to develop its RNA-targeted therapeutics pipeline.

RNA

RNA Drugs Development

Ipsen signs $1.8bn RNA drug alliance with Skyhawk

pharmaphorum

APRIL 22, 2024

bn alliance with Skyhawk Therapeutics focused on RNA-modulating drugs for rare neurological diseases. Ipsen agrees $1.8

RNA

RNA Drugs

Novartis turns to Alnylam in research deal aimed at liver failure

Bio Pharma Dive

JANUARY 6, 2022

The Swiss pharma is well familiar with Alnylam's RNA-based technology, which underpins Novartis' newly approved cholesterol drug Leqvio.

RNA

RNA Research Drugs

Eli Lilly adds ALS drug prospect with QurAlis deal

Bio Pharma Dive

JUNE 3, 2024

The alliance hands Lilly an RNA-based therapy in preclinical testing for ALS and frontotemporal dementia, and includes a research collaboration to develop other, similar medicines.

RNA

RNA Medicine Drugs Development

Ochre Bio raises funds to develop RNA therapies for liver diseases

Pharmaceutical Technology

OCTOBER 11, 2022

Biotechnology company Ochre Bio has raised $30m in a Series A financing round to develop RNA therapies for chronic liver diseases. It also intends to turn the insights from its research into RNA-based drug candidates, and they will be tested in human livers at the company’s recently opened ‘Liver ICUs’ in the US.

RNA

RNA Development Genome Genomics

Daiichi Sankyo taps Depixus to speed RNA drug discovery

pharmaphorum

DECEMBER 13, 2023

Daiichi Sankyo taps Depixus to speed RNA drug discovery Phil.Taylor Wed, 13/12/2023 - 10:43 Bookmark this

RNA

RNA Drugs

Biogen, Ionis tout data from early study of RNA-based Alzheimer's drug

Bio Pharma Dive

JULY 27, 2021

The companies said that patients treated with their experimental drug BIIB080 had reductions in a protein called tau, which research indicates is tied to Alzheimer's but isn't yet proven to impact the course of the disease.

RNA

RNA Drugs Protein Research

FDA grants orphan drug designation to DTx Pharma’s CMT1A therapeutic

Pharmaceutical Technology

JUNE 8, 2023

The US Food Drug Administration (FDA) has granted orphan drug designation to DTx Pharma’s investigational DTx-1252 for the treatment of Charcot-Marie-Tooth disease Type 1A (CMT1A).

Drugs

Drugs Gene Therapy Gene RNA

Alnylam to wait longer for FDA verdict on latest RNA drug

Bio Pharma Dive

APRIL 4, 2022

The FDA has yet to sign off on a third-party packaging and labeling facility that Alnylam planned to help launch its drug, called vutrisiran, meaning an approval decision could come as late as mid-July.

RNA

RNA Packaging Packaging Drugs

FDA denies expanded approval for Alnylam RNA drug

Bio Pharma Dive

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 Drugs Medicine

ReviR Therapeutics partners with Asieris to develop new oncology therapies

Pharmaceutical Technology

FEBRUARY 9, 2023

The collaboration is aimed at discovering and developing new advanced treatments for genitourinary (GU) tumours as well as other related serious diseases using ReviR’s RNA-targeted technologies. ReviR combines computational and high throughput drug discovery technologies to deliver advanced medicines to patients.

Development

Development RNA Gene Therapy Genetics

Alltrna raises $109M for ‘transfer RNA’ drug vision

Bio Pharma Dive

AUGUST 9, 2023

The Flagship-backed company claims its technology can create genetic drugs that work broadly to treat diseases caused by “stop” mutations.

RNA

RNA Drugs Genetics

Pharmas back Accent’s plan to make RNA-targeting cancer drugs

Bio Pharma Dive

JANUARY 23, 2024

J&J and Bristol Myers joined the latest funding round for the startup, which was led by the newly launched Mirae Asset Capital Life Science.

RNA

RNA Life Science Drugs

Moderna’s founder launches Laronde, promising new ‘Endless RNA’ drug class

pharmaphorum

MAY 11, 2021

Flagship Pioneering, the VC fund run by Moderna’s co-founder Noubar Afeyan has launched a new biotech Laronde , with an ambitious plan to create a new class of drugs based on Endless RNA. The post Moderna’s founder launches Laronde, promising new ‘Endless RNA’ drug class appeared first on.

RNA

RNA Drugs Protein Immune Response

Shifting focus, ProQR to seek partners for stalled eye drugs

Bio Pharma Dive

AUGUST 11, 2022

The company will wind down five trials involving two drug candidates and lay off more staff, prioritizing instead its RNA-editing technology.

RNA

RNA Drugs Trials

Comanche Biopharma raises $75M to fund preeclampsia drug testing

Bio Pharma Dive

JANUARY 17, 2024

The biotech is developing an RNA-based therapy for what it describes as a root cause of the pregnancy-related complication, which affects millions of women.

RNA

RNA Drugs Development

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

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

Webinars

Trending Sources

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

Webinars

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

Bayer partners with RNA drugmaker to develop new cancer therapies

Drugging RNA with pills: small molecules for a big frontier

Amgen partners with Arrakis to develop drugs aimed at RNA

China-based biotech licenses Sanofi's RNA drug technology

New UK intracellular drug delivery centre to support RNA development

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

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

AstraZeneca gives up on Ionis’ RNA drug for heart disease

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

Novartis buys a preclinical biotech and its RNA drug technology

AstraZeneca strikes deal with university spinoff to turn RNA into drugs

Roche adds to RNA drug deal streak with Ribometrix partnership

Roche partners with RNA editing biotech Ascidian

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

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

CSL says drug for rare swelling disorder succeeds in late-stage study

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

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

A first-of-its-kind mRNA drug falls short in cystic fibrosis

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

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

KCL study reveals particles in cows’ milk effective for oral delivery of RNA drugs

DCGI adds IVD devices for diagnosis of Covid-19, RNA & DNA extraction kits in Class C risk category under MDR-2017

Tetra and Cellvera partner to develop oral Covid-19 treatment

Daiichi Sankyo and Depixus partner to accelerate RNA-targeted drug discovery

Ipsen signs $1.8bn RNA drug alliance with Skyhawk

Novartis turns to Alnylam in research deal aimed at liver failure

Eli Lilly adds ALS drug prospect with QurAlis deal

Ochre Bio raises funds to develop RNA therapies for liver diseases

Daiichi Sankyo taps Depixus to speed RNA drug discovery

Biogen, Ionis tout data from early study of RNA-based Alzheimer's drug

FDA grants orphan drug designation to DTx Pharma’s CMT1A therapeutic

Alnylam to wait longer for FDA verdict on latest RNA drug

FDA denies expanded approval for Alnylam RNA drug

ReviR Therapeutics partners with Asieris to develop new oncology therapies

Alltrna raises $109M for ‘transfer RNA’ drug vision

Pharmas back Accent’s plan to make RNA-targeting cancer drugs

Moderna’s founder launches Laronde, promising new ‘Endless RNA’ drug class

Shifting focus, ProQR to seek partners for stalled eye drugs

Comanche Biopharma raises $75M to fund preeclampsia drug testing

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