pharmaphorum

OCTOBER 8, 2020

Scribe Therapeutics, a start-up focusing on gene-editing using CRISPR/Cas9, has burst onto the biotech scene with a $415 million deal with Biogen. Further back in development are drugs that will be administered to edit genes within the body, but the first of these candidates are now in clinical trials.

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Gene Editing Gene In-Vivo Clinical Trials 54

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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Gene Editing Trials Drugs Gene 97

pharmaphorum

JUNE 23, 2022

Novartis has shouldered its way into the in vivo gene editing category via a deal with US biotech Precision BioSciences, focused on a therapy for sickle cell disease (SCD). It is also working with Eli Lilly on in vivo gene-editing drugs against three targets, including one in Duchenne muscular dystrophy, under the terms of a $1.4

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Gene Editing In-Vivo Gene Sequencing Gene 81

pharmaphorum

JUNE 28, 2021

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . Intellia and Regeneron’s phase 1 trial looked at patients who had nerve damage (polyneuropathy) as a result of the disease.

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In-Vivo Gene Editing Trials Drugs 92

XTalks

AUGUST 29, 2022

Now a common gene editing tool, the popularity of the CRISPR-Cas9 system has increased over the past decade. CRISPR is notable for engineering living cells, allowing scientists to edit, turn off, delete, or replace genes in a cell’s genome. Harnessing the Cellular Engineering Potential of CRISPR.

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Drug Discovery World

NOVEMBER 14, 2023

A Phase Ib trial has shown low-density lipoprotein cholesterol (LDL-C) reductions up to 55% and blood PCSK9 protein reductions up to 84% after a single infusion of Verve Therapeutics’ Verve-101. It is caused by a single gene mutation that impairs the body’s ability to mediate LDL cholesterol.

In-Vivo 52

In-Vivo Gene Editing Gene Genome 52

WCG Clinical

NOVEMBER 17, 2023

This change expanded the definition of HGT research to include research using nucleic acids that are able to replicate, be transcribed, translated into protein, and/or integrate into the host genome. If implemented, these changes would broaden the definition of HGT to include new gene editing and genome-modified products.

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Genome Genomics Gene Editing Genetic Engineering 52

Drug Discovery World

OCTOBER 10, 2023

The National Institutes of Health has awarded the first phase of a roughly $40m grant to Yale University to advance a novel CRISPR-based gene-editing delivery platform for the targeted treatment of neurogenetic diseases. The two diseases being targeted in the project are Angelman syndrome and H1-4 (HIST1H1E) syndrome.

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Gene Editing Development Drugs Clinical Trials 52

Drug Discovery World

MAY 29, 2024

The project will combine Oxford’s academic research capabilities with SpyBiotech’s proprietary SPYVLP platform technology to advance three vaccine candidates targeting EBV and test these in a Phase I clinical trial. ERS Genomics and IRBM ERS Genomics and IRBM have signed an agreement granting IRBM access to ERS’ CRISPR/Cas9 patent portfolio.

Drugs 52

Drugs Gene Editing Life Science Genome 52

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.

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Gene Editing Gene DNA Engineer 92

Drug Discovery World

MAY 6, 2024

billion on R&D during 2021, with Germany and Switzerland being the biggest contributors, and around one fourth of clinical trials worldwide were performed in Europe 1. The city is host to many innovative biotech companies, including relative newcomers Orexa, a company that recently launched its first Phase II clinical trial.

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Drugs Life Science Gene Editing Gene 59

XTalks

NOVEMBER 3, 2023

CRISPR works as genetic scissors to edit parts of the genome. The CRISPR-Cas9 gene editing system was first discovered to be endogenous in bacteria. Given the system’s powerful ability to make cuts in genes in a precise manner, the system is being leveraged in gene therapies.

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Gene Therapy Gene Gene Editing In-Vivo 59

Drug Discovery World

DECEMBER 12, 2022

In Nature 3 , we learnt about the CRISPR cancer trial success leading the way to personalised treatments. According to the publication, “A small clinical trial has shown that researchers can use CRISPR gene editing to alter immune cells so that they will recognise mutated proteins specific to a person’s tumours.

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Drugs Gene Editing Genome Genomics 52

XTalks

DECEMBER 20, 2023

This includes analyzing how drug combinations may impact individual patients or groups of patients before even entering a clinical trial. With a staggering 90 percent of drugs failing in clinical trials, AI has the potential to help improve these statistics. These companies are at various stages of research and clinical trials.

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Life Science Gene Editing Development Clinical Trials 119

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. These cells present a highly scalable platform, capable of manufacturing advanced cellular therapeutics.

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Gene Editing Gene Engineer DNA 52

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 methods offer safer CAR T-based therapies, laying the foundation for clinical trials and the rapid development of a new generation of CAR T cells for both autologous and allogeneic use.

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DNA Genome Genomics RNA 98

Drug Discovery World

JANUARY 26, 2023

The modernisation of R&D models could be key to reducing spend on new drugs, a point that Deloitte makes about the need to transform the clinical trials process. At the height of the pandemic, DCTs were seen as a viable alternative to keeping the engines running on clinical trials when non-critical in-person care was paused.

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Development Drugs Clinical Trials Clinical Trials 98

pharmaphorum

APRIL 20, 2022

The potential of CRISPR technology has been a hot topic in the industry ever since it was first developed, but as trials progress further into the clinic, what therapeutic areas could be set to benefit? Currently, CAR-T treatments use cells taken from the patient’s own body before editing them and infusing them as a treatment.

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Gene Editing DNA Gene Genetics 52

Drug Discovery World

OCTOBER 25, 2022

CRISPR used to identify genes involved. The scientists used the gene editing technology CRISPR in the lab to break or switch off each of 25,000 genes in cancer cells treated with ATR inhibitors, to identify which genes contributed to drug resistance. . It was funded by Cancer Research UK. .

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Development Drugs Gene Editing Gene 52

XTalks

NOVEMBER 30, 2023

The safety and efficacy of Cabenuva were established through two randomized, open-label, controlled clinical trials involving 1,182 HIV-infected adults who were virologically suppressed (HIV-1 RNA less than 50 copies/mL) before initiating Cabenuva treatment. The Phase I trial for HB-500 is scheduled to start in the first half of 2024.

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FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

The Pharma Data

JANUARY 10, 2021

Nasdaq: EDIT), a leading genome editing company, today announced the U.S. Food and Drug Administration (FDA) has cleared the initiation of the safety phase of the Company’s EDIT-301 clinical trial, and the Company can begin dosing patients. Clinical trial materials are being manufactured by Editas Medicine.

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Medicine Development Clinical Trials Clinical Trials 52

XTalks

FEBRUARY 27, 2024

There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes. The US Food and Drug Administration (FDA) has approved a number of new rare disease drugs in recent years, including gene therapies.

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Research Drugs Gene Therapy Clinical Trials 59

Drug Discovery World

JULY 10, 2023

Artificial intelligence (AI), gene editing, synthetic biology and more, are beginning to prove their usefulness in drug design and development, taking therapies away from the one-size-fits-all approach, towards more definable targets that operate more effectively. There are myriad ways that this is being enabled.

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Gene Therapy Gene Scientist Gene Editing 52

XTalks

MARCH 28, 2024

Early clinical trials have shown promising results and this therapy could become a valuable addition towards CRC treatment in the future. Gene Editing and CAR T-Cell Therapy Genetic engineering technologies, such as CRISPR-Cas9, are providing newer potential avenues for cancer treatment.

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Gene Editing Life Science Clinical Trials Clinical Trials 59

pharmaphorum

AUGUST 31, 2022

In recent trials, researchers found that when the gene therapy was injected into the spinal cords of mice with ALS that SynCav1 protected and preserved spinal cord motor neurons and extended the lifespan of the mice. The list of companies supported includes Moderna, a company focused on mRNA that has also branched into gene editing.

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Gene Therapy Gene Gene Editing DNA 84

Delveinsight

AUGUST 27, 2020

Freenome persists in getting strong financial support for its development work, with USD 270 million in new funds to push its cancer-detecting blood test via clinical trials and across the FDA’s finish line. Freenome secures USD 270 Million to boost its colorectal cancer blood test.

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Vaccination Vaccine Research Trials 53

pharmaphorum

SEPTEMBER 28, 2022

” ArsenalBio’s platform makes use of automation, large-scale genome engineering, using technologies like CRISPR-based gene-editing, and machine learning and artificial intelligence algorithms to design, build, and test cell therapies.

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Gene Editing Genome Genomics Engineer 57

Delveinsight

SEPTEMBER 14, 2021

The outcomes will also be utilized to transition ADARx’s growing pipeline of research phase products into the clinic, initiating Phase 1 clinical trials for ADARx’s lead program. VOR33 comprises CRISPR genome-edited hematopoietic stem and progenitor cells , which have been engineered to exclude CD33.

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RNA Clinical Trials Clinical Trials Drugs 67

Drug Discovery World

DECEMBER 15, 2022

Vor Bio, a clinical-stage cell and genome engineering company, has announced initial clinical data from VBP101, its Phase I/IIa multicentre, open-label, first-in-human study of tremtelectogene empogeditemcel or “trem-cel” (formerly VOR33) in patients with acute myeloid leukemia (AML). . Trem-cel displayed normal engraftment .

Drugs 52

Drugs Genome Genomics Engineer 52

Delveinsight

JANUARY 14, 2021

The companies schedule to initiate a Phase 2 trial assessing combination therapy for both treatment-experienced and treatment-naïve people living with HBV. People in the trial with HBV treatment experience may also receive Gilead’s Vemlidy (tenofovir alafenamide fumarate, TAF). Gilead Sciences, Inc. and Vir Biotechnology, Inc.

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Gene Editing Gene Genetics DNA 40

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.

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Gene Therapy Gene Antibody FDA Approval 59

XTalks

DECEMBER 24, 2020

From isolating SARS-CoV-2 in early January to sequencing its genome shortly thereafter and having a prototype vaccine against it within days, scientific process and progress have held steadfast throughout the pandemic. Infectious disease expert, epidemiologist and director of the Yale Institute for Global Health at Yale University, Saad B.

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Life Science Vaccination Vaccine Gene 111

Drug Discovery World

APRIL 4, 2023

The decision will enable the company to include the US in the global Phase II portion of its ongoing Phase I/II study of NTLA-2002, an in vivo genome editing candidate designed to inactivate the target gene, kallikrein B1 (KLKB1).

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FDA Approval In-Vivo Gene Editing Drugs 52

Pharmaceutical Technology

FEBRUARY 20, 2023

If clinical trial participants are representative of the wider disease population, they can help sponsors assess the effectiveness of new and existing therapeutics. of the clinical trial population globally. participation of white subjects. The technology platforms are almost the same.

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Research Clinical Trials Clinical Trials Trials 130

Drug Discovery World

OCTOBER 16, 2023

Maina Bhaman, Partner at Sofinnova Partners, says: “The UK is an attractive space for drug discovery companies and a prime environment for investment due to its talent, infrastructure, and genomics research. CellProthera also appointed the Newcastle Centre for Life’s GMP cell therapy centre to produce the ATMP.”

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Drugs Life Science Gene Therapy Genome 75

The Pharma Data

JANUARY 24, 2021

With a presidential inauguration and a federal holiday, it wasn’t an enormously busy week for clinical trial news, but there was a fair amount, nonetheless. It inked a deal with the National Institute of Allergy and Infectious Diseases (NIAID) to launch a Phase I trial. Read on to see. COVID-19-Related. Non-COVID-19-Related.

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Trials Allergies In-Vitro Antibody 52

Advarra

NOVEMBER 29, 2022

The first is titled, Human Gene Therapy Products Incorporating Human Genome Editing , and provides recommendations on the information to include in IND filings for these therapies, including study design, safety, and manufacturing. Trend Five: Regulatory Harmonization and Agreements on Surrogate Endpoints.

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