Lilly adds to genetic meds push with Genetic Leap alliance
pharmaphorum
SEPTEMBER 6, 2024
Eli Lilly signs another genetic medicines deal, this time with Genetic Leap, a start-up that applies AI to the discovery of RNA-targeted drugs.
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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.
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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.
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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).
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Silencing RNA causes moles to self-destruct in rare condition
Drug Discovery World
JUNE 20, 2024
UK researchers have designed a new genetic therapy that could alleviate debilitating giant moles in a rare skin condition. The team used a genetic therapy called silencing RNA, which blocks the action of the mutated NRAS in mole skin cells. The treatment could be used to reverse moles, and therefore prevent cancer.
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14th Annual RNA Therapeutics Conference
pharmaphorum
NOVEMBER 1, 2022
14th Annual RNA Therapeutics. Investigating the next generation of genetic medicine through RNA based therapies. RNA therapeutics is a rapidly expanding industry with increasingly growing potential for immunotherapy, personalised medicines, and treatment of genetic, infectious, and chronic diseases.
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RNA interference method could treat muscular dystrophy
Drug Discovery World
DECEMBER 13, 2022
Japanese researchers propose using RNA interference for correcting a mistake in the genetic code of patients with Fukuyama muscular dystrophy. . One form of FCMD is caused by a genetic abnormality in the ‘fukutin’ (FKTN) gene. One form of FCMD is caused by a genetic abnormality in the ‘fukutin’ (FKTN) gene.
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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). DTx-1252 targets the underlying genetic lesion of the disease and leverages our FALCON platform to unlock the promise of RNAi therapeutics. “We
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CAMP4 raises $100m to take lead RNA drugs into clinic
pharmaphorum
JULY 20, 2022
Armed with a $100 million second-round financing, CAMP4 Therapeutics is preparing to start the first clinical trial of a drug targeting regulatory RNA (regRNA) molecules that can be used to fine-tune the expression of genes. ” The post CAMP4 raises $100m to take lead RNA drugs into clinic appeared first on. .
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Junk DNA: How the dark genome is changing RNA therapies
Drug Discovery World
AUGUST 16, 2023
Samir Ounzain , PhD, CEO & Co-Founder of HAYA Therapeutics, looks at how a better understanding of our DNA can lead to increased activity for RNA therapeutics. The whole world realised the power of RNA when the Covid-19 pandemic brought us the first mRNA-based vaccines.
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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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The untapped potential of the transcriptome
Drug Discovery World
JULY 30, 2024
Serna Bio was founded by Rabia Khan , who is also the CEO, to unlock the transcriptome as a drug discovery target. DDW’s Megan Thomas speaks to Khan about the work her team is doing to create the first map of the druggable transcriptome, which involves understanding RNA biology to discover small molecule drugs.
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Genetic biomarkers could personalise therapy for TNBC
Drug Discovery World
AUGUST 16, 2023
A new study has shed light on the genetic messages encoded by genes within ‘triple negative’ breast cancers (TNBC), and shows they could predict response to chemotherapy. Although immune features remained relatively stable, RNA-based features related to DNA damage repair pathways changed significantly.
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Biogen taps Envisagenics’ AI to speed up RNA splicing R&D
pharmaphorum
MAY 14, 2021
Biogen has made a further push into RNA-based drug discovery via a collaboration with Envisagenics, which applies artificial intelligence RNA sequencing data to discover new drug targets and therapeutics. At least 370 genetic disorders are known to derive from errors introduced during this splicing process.
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Potential drug target for difficult-to-treat breast cancer: RNA-binding proteins
Scienmag
JULY 2, 2021
Studies using human cell lines and tumors grown in mice provide early evidence that inhibiting RNA-binding proteins, a previously overlooked family of molecules, might provide a new approach for treating some cancers Credit: UC San Diego Health Sciences In cancer research, it’s a common goal to find something about cancer cells — some sort (..)
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Novo Nordisk and NanoVation enter multi-target partnership
Drug Discovery World
SEPTEMBER 23, 2024
Novo Nordisk and NanoVation Therapeutics have announced a multi-year partnership to advance the development of novel genetic medicines targeting cardiometabolic and rare diseases. Genetic medicine is at a pivotal moment and this partnership marks a major milestone for NanoVation as an innovator in nucleic acid delivery,” said Cullis. “By
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New product could aid successful production of mRNA therapeutics
Drug Discovery World
MAY 21, 2024
Takara Bio has launched PrimeCap T7 RNA Polymerase (low dsRNA), a mutant T7 RNA polymerase suitable for mRNA therapeutic research and development. Further genetic modifications have resulted in a four-fold reduction of cap analogue concentration in the IVT reaction whilst maintaining a capping efficiency above 95%, said the company.
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Genetic profiling of Lyme disease could bring new therapeutic targets
Drug Discovery World
NOVEMBER 17, 2022
The study is the first to use transcriptomics as a blood test to measure RNA levels in patients with long-term Lyme disease. . As part of the study, RNA sequencing was conducted using blood samples from 152 patients with symptoms of post-treatment Lyme disease to measure their immune response. .
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STAT+: Bay Area biotech uses AI to unlock RNA structures — and find new therapies in the process
STAT News
JANUARY 25, 2023
From Covid-19 vaccines to therapies against a range of deadly diseases, every RNA molecule has a complex 3D shape that controls its function. But the startup already has big ambitions for how its AI-based approach can both help biotechs identify RNA-targeting drugs and design RNA to be stable, compact, or to have other desired properties.
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Ginkgo acquires machine learning biotech Patch Biosciences
Drug Discovery World
MARCH 7, 2024
Ginkgo Bioworks has acquired Patch Biosciences, with the intention to strengthen its gene therapy services, cell therapy services, and RNA therapeutics services. JB Michel, CEO and co-founder of Patch Biosciences: “Joining Ginkgo Bioworks represents an exciting step in our journey to engineer better genetic medicines.
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Gene Therapy and Pharmacokinetics
Camargo
JULY 27, 2021
When developing a clinical development program for these increasingly popular therapies, it is important that sponsors use modeling and pharmacokinetic (PK) analysis to evaluate parameters that can be measured while dosing with gene therapy drugs, to characterize exposure-response data and inform rational dosing. Gene Therapy Definition.
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Amvuttra RNAi Therapeutic Wins FDA Approval for Rare Genetic Protein Disorder
XTalks
JUNE 20, 2022
Alnylam Pharmaceuticals, a leading RNA interference (RNAi) therapeutics biopharmaceutical company, announced it received approval from the US Food and Drug Administration (FDA) for its RNAi therapeutic Amvuttra (vutrisiran) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (ATTR) amyloidosis in adults.
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How transcriptomics is driving drug discovery
Drug Discovery World
FEBRUARY 13, 2023
Dr Stephen Barat, Senior Vice President and Therapeutics Division Head at HTG Molecular Diagnostics tells DDW how transcriptomics is driving drug discovery and precision medicines. SB: Transcriptomics is the study of genomic RNA transcripts in a biological system. DDW: Could you explain the process of transcriptomics?
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Solid Biosciences partners with Phlox for cardiac disease therapies
Pharmaceutical Technology
JANUARY 12, 2023
Under the research partnership deal, the companies will develop new precision genetic medicines for a severe form of genetic dilated cardiomyopathy (DCM). We believe the advanced RNA technology and human disease models of Phlox Therapeutics perfectly partner with the delivery tools Solid Biosciences brings to this collaboration.”.
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Lilly and HAYA collaborate to find genome targets for obesity
Drug Discovery World
SEPTEMBER 6, 2024
Eli Lilly and biotechnology company HAYA Therapeutics have announced a multi-year agreement to apply HAYA’s RNA-guided regulatory genome platform to support preclinical drug discovery efforts in obesity and related metabolic conditions.
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smartRNA project joins BII Bio Studio programme
Drug Discovery World
MARCH 21, 2024
A project focused on developing a small RNA-based platform technology for treating monogenic diseases, smartRNA, has joined the BioInnovation Institute’s (BII) Bio Studio programme. The technology has platform potential, as it leverages a universal small RNA pathway to target generic mRNA features.
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This week in drug discovery (13-16 May)
Drug Discovery World
MAY 17, 2024
From a weight loss drug that prevents heart attacks and a gene therapy that restores hearing, to a vaccine that can treat viruses that don’t exist yet, our chosen news stories this week all represent potential breakthroughs in their respective fields. News round-up for 13-16 May by DDW Senior Digital Content Editor Diana Spencer.
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Molecular mechanisms of corona drug candidate Molnupiravir unraveled
The Pharma Data
AUGUST 16, 2021
The antiviral agent incorporates RNA-like building blocks into the RNA genome of the virus. If this genetic material is further replicated, defective RNA copies are produced and the pathogen can no longer spread. Molnupiravir, another antiviral drug candidate, was originally developed to treat influenza.
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Sanofi wagers $400m on miRecule muscular dystrophy therapy
pharmaphorum
OCTOBER 5, 2022
Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRecule. billion deal in 2018.
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A new dawn of the genomic age: five areas set to be transformed in 2023
pharmaphorum
JANUARY 26, 2023
We are already seeing an increase in projects exploring population genomics in Africa, the Middle East, and Asia, with initiatives including the GenomeAsia100K Project and the Genome Aggregation Database focusing on capturing genetic data of non-European individuals. Just one mutation can lead to a complete change in protein formation.
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Next Generation Sequencing (NGS) Library Preparation Kits: Revolutionizing the Field of Genetic Sciences
Roots Analysis
FEBRUARY 23, 2022
Further, NGS library preparation kits offer several benefits, such as low requirement of starting material, generation of longer RNA / DNA sequences and production of superior quality nucleotide libraries, thereby, catering to existing unmet needs of the research domain. Next Generation Sequencing (NGS) Library Preparation Kits. Web: [link].
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Epigenetics discovery could lead to new class of cancer drugs
Drug Discovery World
MARCH 2, 2023
A new paper has solved the 20-year mystery of how epigenetic modifications act as traffic lights to control gene expression and could ultimately speed up the development of a new class of epigenetic cancer drugs. When it gives the green light, H3K4me3 allows RNA polymerase II to move along DNA, transcribing it into RNA as it moves.
Next-generation sequencing and oncology drug discovery
Drug Discovery World
APRIL 4, 2023
Can NGS increase the chances of an oncology drug making it to market? Can NGS identify previously undruggable targets in cancer? How can the technology best be implemented in research labs? Will NGS help drive personalised cancer therapies? Can NGS technology help direct patients towards suitable clinical trials? Register now.
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Small molecule targets SARS-CoV-2 RNA for destruction
Scienmag
SEPTEMBER 30, 2020
Scientists are fighting back with multiple strategies, including vaccines, repurposed drugs developed for other diseases and brand-new therapies. Now, researchers reporting in ACS Central Science have identified small molecules that target a structure within the RNA genome […].
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How next-generation sequencing is informing oncology drug discovery
Drug Discovery World
APRIL 3, 2023
Hosted by DDW and supported by Tecan, “How next-generation sequencing is informing oncology drug discovery” will be free to attend on April 24 at 7AM PST / 10AM EST / 3PM BST / 4PM CET. Join this free webinar to learn about the biggest challenges and opportunities facing the NGS oncology space. You will hear from Darrell Green, a lecturer in (..)
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Free webinar today: How is NGS informing oncology drug discovery?
Drug Discovery World
APRIL 24, 2023
Can NGS increase the chances of an oncology drug making it to market? Can NGS identify previously undruggable targets in cancer? How can the technology best be implemented in research labs? Will NGS help drive personalised cancer therapies? Can NGS technology help direct patients towards suitable clinical trials? Register now.
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52 *Free* Lethal mutagenesis: a viable antiviral strategy with unknown risks
Scienmag
FEBRUARY 3, 2022
In a Perspective, Ronald Swanstrom and Raymond Schinazi argue that antiviral drugs that lead RNA viruses to produce genetic mutations so fast that they cannot remove deleterious ones – a strategy used in antivirals like the recently approved COVID-19 drug molnupiravir – may be viable, but also present unknown risks to the host.
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Webinar: Find out how NGS is informing oncology drug discovery
Drug Discovery World
APRIL 11, 2023
Can NGS increase the chances of an oncology drug making it to market? Can NGS identify previously undruggable targets in cancer? How can the technology best be implemented in research labs? Will NGS help drive personalised cancer therapies? Can NGS technology help direct patients towards suitable clinical trials? Register now.
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Register now to learn how NGS is informing oncology drug discovery
Drug Discovery World
APRIL 18, 2023
Can NGS increase the chances of an oncology drug making it to market? Can NGS identify previously undruggable targets in cancer? How can the technology best be implemented in research labs? Will NGS help drive personalised cancer therapies? Can NGS technology help direct patients towards suitable clinical trials? Register now.
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Can The Next Generation Drug Conjugates Revolutionize The Rare Disorder Domain?
Roots Analysis
OCTOBER 13, 2023
Over the last two decades the pharmaceutical industry has observed a paradigm shift from conventional drug delivery strategies to more enhanced, potent, and targeted therapeutics. This led the researchers to find alternative pathways and drug delivery strategies to enhance the potent delivery of the drug to the site of action.
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Boston Scientific pays $336M; Boehringer Ingelheim acquires Abexxa Biologics; Oncology, RNA biotech 858 Therapeutics inks $60M Series A; Novartis acquires Arctos Medical
Delveinsight
SEPTEMBER 23, 2021
Boehringer Ingelheim acquires Abexxa Biologics for precision most cancers drug pipeline. 858 Therapeutics closes USD 60 Million series A round to drug RNA modulation. Now, they are ready for their next venture, which is launching RNA biotech 858 Therapeutics with a USD 60 million Series A fundraising. Stafford and co.
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Turning science into business: Amplifying mRNA by targeting regRNAs
Drug Discovery World
APRIL 17, 2024
DDW’s Diana Spencer speaks to Josh Mandel-Brehm , CEO of Massachusetts-based biotech CAMP4, to understand the role regulatory RNAs play in gene expression and how the founders overcame the challenge of launching a business based on brand new science. What are the challenges of drug discovery in this area? How do they work?
Folding of SARS-CoV2 genome reveals drug targets — and preparation for ‘SARS-CoV3’
Scienmag
NOVEMBER 20, 2020
Scientists at Goethe University within the international consortium COVID19-NMR refine previous 2D models The genetic code of the SARS-CoV2 virus is exactly 29,902 characters long, strung through a long RNA molecule. It contains the information for the production of 27 proteins.
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Podcast: Innovation drivers and opportunities for drug discovery
Drug Discovery World
AUGUST 1, 2024
This is the latest episode of the free DDW narrated podcast, titled “Innovation drivers and opportunities for drug discovery”, which covers three articles written Volume 24 – Issue 1, Winter 2022/2023 of DDW. The post Podcast: Innovation drivers and opportunities for drug discovery appeared first on Drug Discovery World (DDW).
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