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.”
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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.”
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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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.
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.
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.
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.
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.
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.
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.”
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).
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 →
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.
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.
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).
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.
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.
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.
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
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.
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.
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.
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.,
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.
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).
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.
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.
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%
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.
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.
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.
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
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.
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.
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.
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.
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.
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.
Roots Analysis
AUGUST 31, 2023
It is worth noting that the transcription factor is responsible for recruiting RNA polymerase to bind to the gene and produce messenger RNA, which is then translated into the protein. Overview of Gene Switch The notion that genes might be turned on and off was discovered several decades ago when studies revealed that E.
Drug Discovery World
JANUARY 17, 2024
CTCs reflect high levels of tumour heterogeneity, represent clonal evolution, and are suitable for treatment selection, real-time longitudinal disease monitoring and in vitro/in vivo culture. Brett Swansiger, Chief Commercial Officer at ANGLE discusses the importance of liquid biopsies in precision medicine trials for cancer.
Drug Discovery World
OCTOBER 13, 2022
Prior to the pandemic, messenger RNA (mRNA) drug development was niche and predominantly pointed at different disease targets, including infectious diseases and cancers. Why mRNA has potential . One aspect that makes mRNA technology popular with drug developers is that its proteins can be easily edited. The rise of global demand .
XTalks
APRIL 22, 2022
Flow cytometric receptor occupancy assays are being increasingly used in preclinical and clinical studies. Both the areas of drug development and clinical trials are increasingly using in vitro assays to help determine the efficacy of an investigational therapeutic. What is Flow Cytometry?
Delveinsight
JANUARY 18, 2021
For instance, GTX-102 , an antisense therapy silencing the gene expression by binding to the RNA molecule behind the regulation of its expression, has also secured USFDA Fast Track Designation. Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system. GeneTx and Ultragenyx are co-developing the therapy.
Drug Discovery World
NOVEMBER 13, 2023
Noel Pauli, PhD, Group Leader, Antibody Engineering, Adimab, on: ‘Harnessing in vivo diversities with a yeast-based platform for the discovery of antibodies against multi-pass membrane proteins’. Ali Madani, PhD, Founder and CEO, Profluent Bio, on: ‘Protein engineering with large language models’.
Drug Discovery World
JANUARY 23, 2023
This circumvents several technical challenges inherent to the genetic manipulation of differentiated cells in ex vivo culture or intact tissues. In cell therapy, regenerative and adoptive immunotherapies continue to bridge the gap between genetic engineering strategies and their clinical application.
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