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.
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.
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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.
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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.
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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.
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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.
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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.
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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.
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Exsilio debuts with $82M, pitching a ‘leap’ forward for genetic medicine
Bio Pharma Dive
JUNE 25, 2024
The company claims it can address some of the field’s limitations with medicines delivered via messenger RNA and lipid nanoparticles.
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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.
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Lilly and ProQR to expand genetic medicine development agreement
Pharmaceutical Technology
DECEMBER 23, 2022
Eli Lilly and Company has expanded a licencing and partnership agreement with ProQR Therapeutics to discover, develop and market new genetic medicines. This alliance is utilising the Axiomer ribonucleic acid (RNA) editing platform of ProQR to address ailments affecting the liver and nervous system.
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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.
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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.
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Biology opens up new frontiers in medicine to cure genetic diseases: Kiran Mazumdar Shaw
AuroBlog - Aurous Healthcare Clinical Trials blog
SEPTEMBER 17, 2024
Biology is opening up new frontiers in medicine. We are witnessing a revolution in healthcare, driven by advances in genetics, Omics, RNA and CRISPR gene-editing technology, to deliver precision and personalised medicine, said Kiran Mazumdar-Shaw, executive chairperson, Biocon and Biocon Biologics.
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The critical role of comprehensive RNA sequencing in liquid biopsy for biomarker discovery and clinical trials
Bio Pharma Dive
JUNE 17, 2024
Elevate precision medicine: dose optimization and immune monitoring through advanced liquid biopsies.
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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.
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Vertex tiptoes further into Duchenne research with Tevard deal
Bio Pharma Dive
FEBRUARY 28, 2023
An alliance with the startup brings Vertex an RNA-based medicine for the disease, adding to a portfolio that already includes a gene editing treatment.
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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.
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State of Play: A closer look at hotspots of emerging biotech research
Bio Pharma Dive
NOVEMBER 29, 2022
In this continuing series, BioPharma Dive examines new areas of biotech investment and startup activity, such as next-generation RNA medicines, TYK2 inhibitors and in vivo CAR-T.
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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.
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GSK dives into NASH, RNA interference with Arrowhead deal
Bio Pharma Dive
NOVEMBER 23, 2021
GSK has secured rights to a genetic medicine in early-stage human testing for NASH, a disease thought to affect millions of people and which has proven to be a tough target for drugmakers.
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Merck and Orna partner for RNA technology-based vaccines and therapies
Pharmaceutical Technology
AUGUST 17, 2022
Merck (MSD outside North America) has entered a partnership agreement with Orna Therapeutics for discovering, developing and marketing various programmes based on next-generation RNA technology. By self-circularisation, Orna’s oRNA technology makes circular ribonucleic acids (oRNAs) from linear RNAs.
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Biotech investors, including Third Rock, pump $100M into a startup’s genetic medicine for ALS
Bio Pharma Dive
NOVEMBER 12, 2024
Trace Neuroscience aims to advance an RNA-binding therapy designed to preserve — and potentially improve — muscle function in people living with the nerve-destroying disorder.
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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.
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Arrowhead reports positive data from liver drug, but company’s shares slide
Bio Pharma Dive
JANUARY 9, 2023
The biotech and partner Takeda also announced a Phase 3 study of the RNA medicine that is longer than anticipated, pushing out analysts’ timelines.
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Alnylam becomes new partner in the Medicines Manufacturing Innovation Centre
Pharma Times
OCTOBER 7, 2021
Leading RNA intereference (RNAi) therapeutics company, Alnylam, has been welcomed as the latest partner in the Medicines Manufacturing Innovation Centre, to transform the manufacturing process of oligonucleotide therapies.
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Switch Therapeutics launches with $52M to capitalize on RNA medicine ‘era’
Bio Pharma Dive
MARCH 14, 2023
The company’s name comes from its goal of developing drugs that only “switch” on in target cells, potentially enabling more precise therapies.
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Pushing Beyond Gene Editing Limits with Programmable RNA Medicines
BioSpace
JUNE 12, 2022
Shape Therapeutics is evolving programmable RNA medicines that push the limits of current gene editing capabilities. BioSpace spoke with CSO David Huss.
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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.
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ReNAgade Launches with $300M to More Easily Deliver RNA Medicines
BioSpace
MAY 22, 2023
ReNAgade Launches with $300M to More Easily Deliver RNA Medicines 5/23/2023
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Locanabio’s RNA-Targeting Gene Therapy May Offer Durable Response for DM1 Patients
BioSpace
SEPTEMBER 22, 2020
RNA-binding proteins are emerging as the next generation of molecular medicine.
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Deep Genomics is Rewriting RNA for Powerful Therapeutics with $180M Series C
BioSpace
JULY 27, 2021
"For the first time in history, medicine has become information." That's the groundbreaking connection AI-driven RNA therapeutics has given us, according to Deep Genomics Founder and CEO Brendan Frey.
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Ionis teams with Metagenomi and dives into gene editing
Bio Pharma Dive
NOVEMBER 14, 2022
The RNA drug developer is paying the richly-funded startup $80 million in a wide-ranging alliance that marks its first foray into DNA editing medicines.
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Chinook and Ionis partner to develop ASO therapy for kidney disease
Pharmaceutical Technology
MAY 17, 2023
It will use the precision medicine approach, as well as the deep expertise of Chinook Therapeutics in nephrology and knowledge of Ionis Pharmaceuticals in RNA-targeted therapeutics. The collaboration aims to discover, develop and commercialise an ASO therapy.
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Jupiter Neurosciences Makes Way in CNS Treatments with $11M IPO
XTalks
DECEMBER 4, 2024
In the Asia-Pacific market, Jupiter has negotiated partnerships with companies such as Sichuan Kelun and Tianjin Pharmaceuticals, focusing on Jotrol’s integration into traditional Chinese medicine frameworks. International patents valid until 2036 further strengthen its global positioning.
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STAT+: After blazing trails in RNA-targeted drugs, Ionis jumps into gene editing
STAT News
NOVEMBER 14, 2022
Ionis Pharmaceuticals, the California company that turned RNA-targeting medicines from an unproven idea into blockbuster drugs, is now expanding into a new class of therapeutics that treat disease by editing DNA. On Monday, the company announced a partnership with Metagenomi to kickstart these efforts.
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RNA sequencing of amniotic fluid cells for prenatal diagnosis
Medical Xpress
DECEMBER 29, 2022
A clinical research team from the University of Hong Kong (HKUMed) has used amniotic fluid cells obtained during 16-24 weeks of pregnancy as a novel sample type for RNA-sequencing in prenatal diagnosis to help more families with tailored clinical management. The findings have been published in the journal, npj Genomic Medicine.
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Scientists develop an RNA-based breath test to detect COVID-19
Scienmag
DECEMBER 13, 2021
Philadelphia, December 13, 2021 – In a new study in The Journal of Molecular Diagnostics, published by Elsevier, investigators report on the design and testing of a breathalyzer, known as the Bubbler, that relies on viral RNA detection to diagnose SARS-CoV-2.
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Double-stranded RNA induces bone loss during gum disease
Scienmag
APRIL 8, 2022
They found that double stranded RNA molecules can activate the immune system response that leads to deterioration of bone. Tokyo University of Agriculture and Technology researchers reported on a new discovery regarding the mechanisms for bone loss in gum disease (periodontitis).
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Lilly buys into MiNA’s protein-boosting RNA tech in $1.25bn deal
pharmaphorum
MAY 12, 2021
UK biotech MiNA Therapeutics has signed up another big pharma partner for its small activating RNA (saRNA) platform, which upregulates the activity of proteins, with Eli Lilly the latest to get in on the action. . The post Lilly buys into MiNA’s protein-boosting RNA tech in $1.25bn deal appeared first on.
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Agios turns to Alnylam for help with rare disease push
Bio Pharma Dive
AUGUST 3, 2023
The biotech is acquiring rights to an RNA interference medicine Alnylam invented for a rare blood disorder, continuing a corporate makeover that began in 2020.
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Scientists develop RNA-targeting strategy to repair genetic cause of ALS and dementia
Medical Xpress
NOVEMBER 22, 2022
Scientists at University of Florida (UF) Scripps Biomedical Research have developed a potential medicine for a leading cause of ALS and dementia that works by eliminating disease-causing segments of RNA. The compound restored the health of neurons in the lab and rescued mice with the disease.
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