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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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.
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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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.
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.
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.
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.
Pharmaceutical Technology
MAY 23, 2023
ReNAgade Therapeutics has burst onto the RNA technology scene by announcing a $300m financing round. With its delivery system, Cambridge, Massachusetts-based biotech says it aims to “address major limitations in RNA therapeutics by enabling the delivery of RNA medicines to previously inaccessible tissues and cells in the body.”
Pharmaceutical Technology
MAY 25, 2023
ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. The technology platforms include the Life Edit gene editing platform, an RNA, cell, protein, vector engineering and induced pluripotent stem cells (iPSCs) platform.
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.
The Pharma Data
JUNE 7, 2023
Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., This delivery technology protects the messenger RNA (mRNA) payload after administration allowing it to be safely and effectively delivered into cells.
Drug Discovery World
OCTOBER 14, 2022
Meanwhile, Ochre Bio successfully raised $30 million to advance its pipeline of RNA therapies for liver disease, and researchers found that combining CRISPR gene editing with Spherical Nucleic Acids could expand the potential of the technology. . 30 million financing for RNA therapies for chronic liver diseases .
Drug Discovery World
JUNE 22, 2023
“This is part of our long-term business strategy of leveraging exosome-mediated delivery to create and enable genetic medicines such as gene therapy and genome editors”.
The Pharma Data
MARCH 7, 2022
By combining CRISPR technology with a protein designed with artificial intelligence (AI), it is possible to awaken individual dormant genes by disabling the chemical “off switches” that silence them. Researchers from the University of Washington School of Medicine in Seattle describe this finding in the journal Cell Reports.
Pharma Marketing Network
DECEMBER 21, 2020
Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., 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.
Drug Discovery World
DECEMBER 21, 2023
In a record year for gene therapy approvals, it isn’t surprising that cell & gene therapy (CGT) has been a hot topic. Other biotechnologies coming to the fore this year were antibody-drug conjugates and CRISPR gene editing. This well-read article featured in the DDW Winter 22/23 issue’s Cell & Gene Therapy Guide.
The Pharma Data
DECEMBER 5, 2020
5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Both of the new gene therapy studies were published online Dec. 5 in the New England Journal of Medicine. SATURDAY, Dec.
Drug Discovery World
NOVEMBER 7, 2022
Genomic healthcare data is critical to identify disease risk, ancestry, traits and response to medicines and aids in the development of new targeted therapies – precision medicines. DDW’s Megan Thomas observes developments of accessibility in this sector and the potential impact. . The origins . The current landscape .
The Pharma Data
DECEMBER 13, 2020
“Our company and R&D portfolio are entering into an exciting phase, as evidenced by the recent close of Series B financing and submission of the first gene editing product IND in China,” said Dong Wei, Ph.D.?CEO Prior to joining EdiGene, he was Vice President of KLUS Pharma and focused on cell therapy and new technologies.
Delveinsight
SEPTEMBER 14, 2021
ADARx bags USD 75 Million to advance its RNA tech pipeline. ADARx Pharmaceuticals, a biotechnology company developing RNA targeting therapeutics , announced the completion of a USD 75 million Series B financing to progress its drug development pipeline. SR One Capital Management and OrbiMed Advisors co-led Series B. Simeon George, M.D.,
Drug Discovery World
DECEMBER 15, 2022
As gene editing technologies like CRISPR progress toward clinical study, researchers must continue to advance new approaches and address inherent challenges, explains Jon Chesnut, PhD, Senior Director, Cell Biology R&D, Thermo Fisher Scientific. Early phase clinical trials for gene editing therapies.
Delveinsight
FEBRUARY 25, 2021
Beam Therapeutics has taken over a startup whose technology could proffer the biotech’s genetic medicines to more tissues in the body, widening the potential to approach more diseases. The gene-editing technologies, which have reached the clinic, CRISPR, zinc finger nucleases, and TALENs, make their edits by nicking DNA at the target site.
Drug Discovery World
OCTOBER 16, 2023
A company within this ecosystem is STORM Therapeutics, a clinical stage biotechnology company creating novel small molecule therapies that inhibit RNA modifying enzymes (RME) for use in oncology and other diseases. billion in funding. Government investment of £1.3
Drug Discovery World
MARCH 11, 2024
Researchers from Japan have used CRISPR gene editing to enhance the effectiveness of rejuvenated cytotoxic T lymphocytes (rejTs) for targeted immunotherapy. To overcome this issue, the team used CRISPR-Cas9 two-step ‘scarless’ gene editing on iPSCs derived from an HPV-specific cytotoxic T lymphocyte clone.
Bioengineer
JULY 23, 2021
Jain also has a joint appointment in the College of Medicine at Texas A&M. The team also works with Dr. Gang Bao, a gene editing expert from Rice University. “For the first time, we identified a crucial interaction between platelets and the tumor via their surface proteins,” Jain said. ” ###.
pharmaphorum
FEBRUARY 3, 2021
Dr Jennifer Harbottle, senior scientist in the R&D Base Editing team of PerkinElmer’s Horizon Discovery business, looks at progress made in the realms of biotechnology and next-generation diagnostics, vaccines and therapeutics, including the application of CRISPR-Cas9 gene editing in developing and refining cell therapies.
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. Other CRISPR-based therapies generate a DSB at a precise location, after which cellular processes carry out the editing function.
XTalks
DECEMBER 22, 2021
The RNA Revolution: From mRNA Vaccines to RNA Editing. The age of RNA is officially here, and it’s here to stay as more than a passing life science trend. RNA technology is not new nor has its potential been surprising. RNA in the Making. So why did this perceived RNA ‘revolution’ take so long?
pharmaphorum
JUNE 28, 2021
The reductions matched the efficacy of current therapies for ATTR amyloidosis that require chronic dosing such as Alnylam’s Onpattro (patisiran) and Ionis/Akcea’s Tegsedi (inotersen) – both gene-silencing agents which can cost around $450,000 a year. — Eric Topol (@EricTopol) June 26, 2021.
pharmaphorum
JANUARY 10, 2022
Previously, messenger RNA (mRNA) therapies were a niche part of the global R&D pipeline, now a wide section of the public is at least familiar with the name of this type of therapy. The biotech is focused on mRNA but has developed a self-amplifying mRNA (sa-RNA) platform in an attempt to develop the potential of the technology further.
The Pharma Data
JANUARY 19, 2021
A naturally occurring system for tuning CRISPR-Cas9 expressing in bacteria, identified in a study published in Cell , could have implications for gene editing therapies as well. A CRISPR-Cas9 system has two components: the Cas9 guide RNA that directs the system to edit a particular gene, and the CRISPR “scissors” that make the cut.
Drug Discovery World
JANUARY 23, 2023
Kevin Hemphill, R&D Manager at PerkinElmer’s Horizon Discovery explores how base editing has emerged as an attractive gene editing option for researchers wanting to develop stem cell-based therapies. Adult fibroblasts were cultured in a cocktail of transcription factors to reactivate embryonic gene expression patterns 2.
Drug Discovery World
JULY 18, 2023
Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. These efforts, along with many others, are opening doors for new possibilities in translational medicine. parvovirus B19, Flaviviridae, Ebola, and Coronaviridae), DNA viruses (e.g.,
The Pharma Data
NOVEMBER 22, 2020
Scientists in Israel have used the CRISPR Cas-9 gene editing system to destroy cancerous cells in mice without damaging other cells. The product is the most advanced gene-editing approach in development for transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease. Photo courtesy of Science Advances.
Drug Discovery World
MAY 30, 2023
From antibody-drug conjugates and RNA, to CRISPR and CAR-T therapy, DDW asked the drug discovery industry what the most important development in oncology over the last five years has been. One of the most exciting recent developments is the use of gene editing tools to create cell and gene therapies.
XTalks
SEPTEMBER 21, 2020
UCSF scientists found that having an additional copy of the sex chromosome gives women two “doses” of a gene found only on that chromosome. The study was published in the journal Science Translational Medicine. The gene, called KDM6A , is a histone demethylase that is believed to function as a tumor suppressor. The Active Gene.
XTalks
AUGUST 2, 2023
With each passing year, pharmaceutical companies around the globe strive to deliver cutting-edge medicines, therapies and vaccines that impact the lives of millions. In this in-depth article, we embark on a captivating journey to uncover the top 30 pharma and biotech companies that have been instrumental in shaping the future of medicine.
Drug Discovery World
FEBRUARY 22, 2024
According to Medicine Discovery Catapult, these include: “Navigating the complexity of the CNS; understanding the disease pathology, and accessing predictable in vitro and in vivo cell models 2 ”. We should not overlook neuropsychiatric conditions such as depression, anxiety and addiction either.
XTalks
DECEMBER 24, 2020
From rare disease drug approvals to treatments involving immunotherapies and gene therapies and awarding of a Nobel Prize to the inventors of the gene-editing tool CRISPR, 2020 was a year of great activity and productivity despite the backdrop of the pandemic. CRISPR Gene Editing Inventors Win Nobel Prize.
Pharmaceutical Technology
FEBRUARY 20, 2023
He says these issues must be urgently addressed over the next few years as advancing medical knowledge and the role of genomics in treating disease ushers in a new era of precision medicine and new potential to address diseases that affect certain populations. The technology platforms are almost the same.
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