Moderna inks another gene editing deal
Bio Pharma Dive
FEBRUARY 22, 2023
The messenger RNA specialist said Wednesday it is teaming up with Life Edit Therapeutics to develop therapies that can modify genes “in vivo.”
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.
In-Vivo 
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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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How does circVec technology compare to conventional mRNA expression?
Drug Discovery World
JULY 3, 2024
Circio has announced updated in vivo data that demonstrates a substantial durability advantage of Circio’s circVec technology over conventional mRNA expression. design is performing very well in vivo , and Circio has now validated expression for up to five months. design. “The circVec 2.1
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Circio debuts proof-of-concept for circVec gene therapy at ASGCT 2024
BioPharma Reporter
MAY 13, 2024
Circio, a biotech firm specializing in circular RNA-based gene therapy, has unveiled two posters showcasing in vivo proof-of-concept for its circVec platform at ASGCT 2024.
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EdiGene Biotechnology moves into new R&D center in Massachusetts
BioPharma Reporter
NOVEMBER 10, 2022
to advance its proprietary LEAPER tech into in vivo RNA editing therapies: with an initial focus on ophthalmology and the central nervous system. EdiGene Biotechnology USA has moved into a new Research & Development Center in Waltham, Mass.
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Myeloid raises funds to support lead cell therapy programme
Pharmaceutical Technology
MAY 19, 2023
MT-302 is a TROP2-targeting in vivo chimeric antigen receptor (CAR) that has been designed to express in the myeloid compartment. The financing will help to fast-track the development of other in vivo programming candidates into clinical trials. Topic sponsors are not involved in the creation of editorial content.
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Moderna seals pact with Life Edit Therapeutics to tackle genetic diseases with mRNA-based gene editing
BioPharma Reporter
FEBRUARY 23, 2023
The messenger RNA (mRNA) specialist Moderna has teamed up with ElevateBio-owned Life Edit Therapeutics to develop gene editing therapies that are delivered into patients in vivo.
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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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Moderna partners with Life Edit for mRNA gene editing therapies
Pharmaceutical Technology
FEBRUARY 23, 2023
Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Our novel editing systems have the potential to precisely modify gene targets for both in vivo and ex vivo therapeutic development.”
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Conversations from ESMO Targeted Anticancer Therapies Congress 2024
Drug Discovery World
MARCH 26, 2024
Additional approaches, such as profiling of RNA and proteins, and the use of artificial intelligence (AI), could be key to providing a more comprehensive picture of a patient’s tumour and enable the use of optimised treatments. MT: Why was STORM attending the conference? We were there to present the latest findings from our research.
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Shining a light on the role of the genome’s ‘dark matter’ in cancer development
Scienmag
NOVEMBER 16, 2020
Singapore scientists uncover potential role of long non-coding RNAs in pancreatic cancer Credit: From Figure 4 in Liu, S., Wnt-regulated lncRNA discovery enhanced by in vivo identification and CRISPRi functional validation. Harmston, N., Glaser, T.L. Genome Med 12, 89 (2020).
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This week in drug discovery (13-16 May)
Drug Discovery World
MAY 17, 2024
Data demonstrates superiority of circular RNA vs linear mRNA In vivo proof-of-concept for Circio Holding’s differentiated circVec platform approach to gene therapy has been demonstrated in two posters at the American Society of Gene & Cell Therapy (ASGCT) 2024 annual meeting.
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Wave Life Sciences Announces Proof-of-Concept Preclinical Data for ADAR Editing Program in Alpha-1 Antitrypsin Deficiency
BioTech 365
JUNE 2, 2021
Wave Life Sciences Announces Proof-of-Concept Preclinical Data for ADAR Editing Program in Alpha-1 Antitrypsin Deficiency Wave Life Sciences Announces Proof-of-Concept Preclinical Data for ADAR Editing Program in Alpha-1 Antitrypsin Deficiency First proof-of-concept in vivo data for RNA editing using endogenous … Continue reading →
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Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics
The Pharma Data
JUNE 7, 2023
Acuitas’ LNP technology will support Bayer’s in vivo gene editing and protein replacement programs by specifically delivering RNA payloads to the desired target organ, the liver. a biotechnology company specializing in the development of lipid nanoparticle (LNP) delivery systems for molecular therapeutics.
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Leading innovators in mammalian expression vectors for the pharmaceutical industry
Pharmaceutical Technology
FEBRUARY 14, 2023
Innovation S-curve for the pharmaceutical industry Mammalian expression vectors is a key innovation area in pharmaceutical Mammalian expression vectors are used to introduce a specific fragment of DNA into mammalian systems for RNA or protein expression. These systems include human and mouse cell lines, and even in vivo in live animals.
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RNA companies to watch over the next 12 months
Drug Discovery World
JANUARY 23, 2023
Following the release of Clarivate’s report ‘RNA Technology Companies to Watch’, DDW’s Diana Spencer speaks to Strand Therapeutics’ Jacob Becraft and HAYA Therapeutics’ Samir Ounzain to find out how this technology could revolutionise disease treatment.
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Spotlighting Lupus Awareness Month: CAR-T Technology Creates New Avenues for Treatment of a Devastating Disease
WCG Clinical
MAY 28, 2024
The currently approved CD-19 CAR products are autologous, ex vivo cellular therapies. Recent headlines have highlighted the potential for Chimeric Antigen Receptor (CAR)-based therapies to provide clinical benefits to persons affected by lupus. In these cases B cell depletion is a feature, not a bug.
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LNP formulations produce strong immune responses, data shows
Drug Discovery World
FEBRUARY 15, 2024
The etherna paradigm for cLNP design is ushering in the rapid generation of novel families of delivery systems for not only vaccination but also for in vivo tolerisation for a host of autoimmune diseases.” The post LNP formulations produce strong immune responses, data shows appeared first on Drug Discovery World (DDW).
Data demonstrates superiority of circular RNA vs linear mRNA
Drug Discovery World
MAY 15, 2024
In vivo proof-of-concept for Circio Holding’s differentiated circVec platform approach to gene therapy has been demonstrated in two posters at the American Society of Gene & Cell Therapy (ASGCT) 2024 annual meeting. The in vivo data showed statistically significant higher expression level and durability for circVec 2.1
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Combining the Powers of Single Cell Sequencing and AI in Understanding Disease Biology and Drug Development
XTalks
AUGUST 5, 2020
In recent webinars by Genuity Science, formerly known as WuXi NextCODE, experts from the biotech and pharma industries spoke about leveraging the power of single cell RNA sequencing platforms and solutions in conjunction with machine learning technologies such as AI in cell biology and disease research. The Power of Single Cell Technology.
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FDA-approved drug sensitises brain cancer cells to radiotherapy
Drug Discovery World
JANUARY 23, 2023
This action sensitised GBM cells to radiotherapy both in vitro and in vivo (in mouse models). “Our research has revealed cladribine as a radiosensitiser for GBM treatment by drug repurposing, which can offer multiple advantages,” says Prof Youn. “As GBM is a WHO grade IV brain tumour with dismal prognosis.
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Preliminary Findings from a Phase 2a Trial of Investigational COVID-19 Therapeutic Molnupiravir
The Pharma Data
MARCH 8, 2021
This program included assays such as Big Blue and PIG-a designed to provide a robust measure of a drug or chemical’s ability to induce mutations in vivo. The totality of the data from these studies indicates that molnupiravir is not mutagenic or genotoxic in in vivo mammalian systems. “We
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AACR: A focal point of the cancer research community
Drug Discovery World
JULY 15, 2024
Sapetschnig said that STORM will continue to develop a pipeline of therapies inhibiting RNA modifying enzymes and advance this candidate towards clinical development. From 5-10 April 2024 in San Diego, the cancer research community gathered for the annual meeting of the American Association for Cancer Research (AACR).
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EdiGene Expands Management Team by Appointment of Head of US Subsidiary Dr. Bo Zhang and Head of Business Development Dr. Kehua Fan
The Pharma Data
DECEMBER 13, 2020
The company has established its proprietary ex vivo genome-editing platforms for hematopoietic stem cells and T cells, in vivo therapeutic platform based on RNA base editing, and high-throughput genome-editing screening to discover novel targeted therapies.
Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem
Pharma Marketing Network
DECEMBER 21, 2020
those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies. Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e.,
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RNA editing oligonucleotide selected for AATD treatment
Drug Discovery World
DECEMBER 12, 2023
KRRO-110 is designed to co-opt an endogenous enzyme Adenosine Deaminase Acting on RNA (ADAR) to repair a pathogenic single nucleotide variant (SNV) on RNA and restore production of normal A1AT.
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Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug
pharmaphorum
JUNE 28, 2021
So far, human trial data for gene-editing drugs have been generated for ex vivo therapies, meaning the CRISPR technology is used to modify cells outside the body, which are then reinfused into the patient. — Eric Topol (@EricTopol) June 26, 2021. — Eric Topol (@EricTopol) June 26, 2021.
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What the Glycome Can Tell Us About Persistent HIV Infection
XTalks
AUGUST 12, 2020
Studies have identified the presence of two types of HIV-infected CD4+ T cells: ‘Transcriptionally inactive’ cells that do not typically produce viral RNA or viral proteins. Transcriptionally active’ cells in which HIV RNA is actively transcribed to make copies of the virus (despite long-term ART).
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Beam makes USD 120M bet; GRAIL and Quest announce the collaboration; Brain organoids mimic infant’s brains; Improvement in T cells to kill cancer
Delveinsight
FEBRUARY 25, 2021
Electroporation is used for ex vivo delivery of therapies to blood and immune cells. The company’s in vivo therapies reach the eye and central nervous system via adeno-associated viruses, while its liver therapies employ lipid nanoparticles (LNPs). Beam employs three approaches to deliver its genetic medicines to cells.
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Cell-penetrating peptides as a delivery system for oligonucleotides
Drug Discovery World
AUGUST 3, 2022
Oligonucleotides act on the RNA level through different molecular pathways, but there is a major drawback when using them as drugs: their poor bioavailability and cellular uptake. This method is generally used to deliver small interfering RNA (siRNA), but it can also be used for larger oligonucleotides too. Oligonucleotide. Anti-miRNA.
Taking an ‘upside-down’ approach to mRNA delivery
pharmaphorum
JANUARY 17, 2023
The intracellular barriers include endosomal escape, RNA sensors, and endonucleases. Ben Hargreaves speaks to Liberate Bio, a company that is hoping to address the delivery issues that could prevent mRNA, as well as siRNA and ASO therapeutics, from reaching their full potential.
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Drug discovery quarterly review: Second quarter 2023
Drug Discovery World
SEPTEMBER 14, 2023
Moreover, in July 2023, AstraZeneca and Vaxess Technologies commenced a collaboration for the evaluation of a novel RNA-based pandemic influenza prototype vaccine in patch format. Business updates Pfizer reported second quarter 2023 revenues totalling $12.7 billion, a decrease of $15.0 Vaccines were up 9.1%
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China NMPA grants IND clearance for Ascletis’ Covid-19 therapy
Pharmaceutical Technology
AUGUST 21, 2022
An RNA-dependent RNA polymerase (RdRp) inhibitor, ASC10 is an oral double prodrug. D-N4-hydroxycytidine (NHC) in vivo. The China National Medical Products Administration (NMPA) has granted clearance for Ascletis Pharma’s Investigational New Drug (IND) application of its drug candidate, ASC10, for Covid-19.
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Positive preclinical data for siRNA therapy for osteopetrosis
Drug Discovery World
SEPTEMBER 25, 2023
RNA therapeutics company SiSaf has published positive safety and efficacy data for its siRNA therapy for Autosomal Dominant Osteopetrosis 2 (ADO2). Co-author on the paper, Dr Suzanne Saffie-Siebert, CEO of SiSaf, said: “Targeting the bone safely and effectively is a challenge for RNA-based therapies.
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Multispecific antibody shows promise in B cell lymphoma
Drug Discovery World
JUNE 17, 2024
One dose of Nutcracker Therapeutics’ NTX-472 in vivo rapidly depleted B cells with no detectable binding to red blood cells. We’re proud to be one of the first RNA therapeutics companies to engineer a multispecific antibody,” said Chief Scientific Officer Samuel Deutsch. “We
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How AI in pharma can live up to the hype
Drug Discovery World
SEPTEMBER 28, 2022
Artificial intelligence (AI) is notorious for its industry hype, touted to revolutionise the way multiple sectors operate over the next five to 10 years. Its application in pharma, most commonly in drug discovery, has been steadily growing with global partnerships between tech vendors and pharma companies becoming more commonplace.
The challenges and trends of cell & gene therapies
Drug Discovery World
SEPTEMBER 6, 2022
This area of medicine, which turns our own bodies into agents of combat to fight disease, has quickly become one of the most promising fields in treating deadly diseases such as cancer, central nervous system disorders and even musculoskeletal conditions. . Last year saw the sector surge, with levels of investment hitting an all-time high at $22.7
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Organ-on-a-chip models in drug development
Drug Discovery World
APRIL 12, 2023
Existing in vivo and in vitro models aren’t up to the challenge Preclinical tools exist for the evaluation and screening of potential drug candidates. Existing in vivo and in vitro models aren’t up to the challenge Preclinical tools exist for the evaluation and screening of potential drug candidates.
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Organ-on-a-chip models in drug development
Drug Discovery World
JANUARY 20, 2023
Existing in vivo and in vitro models aren’t up to the challenge Preclinical tools exist for the evaluation and screening of potential drug candidates. Existing in vivo and in vitro models aren’t up to the challenge Preclinical tools exist for the evaluation and screening of potential drug candidates.
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ImmunityBio’s hAd5 T-Cell COVID-19 Vaccine Candidate Shows Complete Protection of Airways in Non-Human Primates
The Pharma Data
DECEMBER 10, 2020
.–( BUSINESS WIRE )– ImmunityBio , a privately-held immunotherapy company, today announced its COVID-19 vaccine candidate protected nasal and lung airways of non-human primates against coronavirus (SARS-CoV-2) in a challenge study. This blocking of viral replication was observed in both the lung and nasal passages.
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CRISPR breakthroughs: New solutions for common diseases
Drug Discovery World
JULY 18, 2023
Incorporating in vivo pooled CRISPR screens has allowed for the interrogation of thousands of genetic perturbations in cells, leading to the determination of the involvement of molecules (PTPN2, ADAR1, or DHX37) in immune checkpoint inhibitor (ICI) responsiveness and T cell activation.
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Delivering on the promise of gene editing
Drug Discovery World
DECEMBER 15, 2022
Prime editing, while also employing a single strand nick, expands this scope to all 12 possible changes using a more complex prime editing guide RNA (pegRNA). Improve delivery: Clinical translation of gene therapy is dependent on the efficient and safe delivery of gene editing tools into cells, and this can occur either ex vivo or in vivo.
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Verve starts trials of cholesterol drug in test of base editing technique
pharmaphorum
JULY 13, 2022
Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment. dosing of *first patient* with VERVE-101, an in vivo CRISPR base editing medicine. Today: we are announcing.
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