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.

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Gene Editing DNA Gene Genome 98

pharmaphorum

OCTOBER 7, 2020

Drs Emmanuelle Charpentier and Jennifer Doudna have won this year’s Nobel Prize for chemistry in recognition of their work on the gene-editing technology CRISPR/Cas9. There is enormous power in this genetic tool, which affects us all. What started as a curiosity?driven, million) Nobel Prize award. “In

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Gene Editing Bacteria DNA Gene 105

Pharmaceutical Technology

MARCH 2, 2023

Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. The therapeutic candidate comprises allogeneic NK cells genetically engineered to express chimeric antigen receptors (CAR-NK) targeting cells CD33.

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Gene Editing Genetic Engineering Engineer Clinical Trials 100

Pharmaceutical Technology

MARCH 2, 2023

Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. The therapeutic candidate (CAR-NK) comprises allogeneic genetically modified NK cells expressing chimeric antigen receptors (CAR) targeting cells expressing B lymphocyte antigen CD19.

Gene Editing 100

Gene Editing Clinical Trials Clinical Trials Genetics 100

XTalks

JANUARY 26, 2024

Innovations in Cancer Therapy CRISPR/Cas9, a groundbreaking gene-editing technology, has demonstrated significant potential in oncology, offering new avenues for cancer treatment. By tailoring treatments based on an individual’s genetic makeup, it may allow for more effective and targeted therapies.

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Research Gene Editing Gene Genetics 118

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.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

In-Vivo 52

In-Vivo Gene Editing Trials Gene 52

pharmaphorum

FEBRUARY 23, 2022

In this context, the 2 nd Annual Gene Therapy Patient Engagement Summit will unite patient advocacy and engagement leaders from gene therapy and gene editing drug developers, patient groups and patients themselves to address the specific challenges of genetic therapy patient engagement, generating actionable insights through detailed case studies (..)

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Gene Therapy Gene Gene Editing Clinical Trials 52

pharmaphorum

JANUARY 10, 2022

Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences.

In-Vivo 52

In-Vivo Gene Editing Gene Therapy Gene 52

WCG Clinical

NOVEMBER 17, 2023

Since then, however, certain genetic engineering technologies (e.g., CRISPR-based gene editing) have advanced to the point where cellular genomes can be edited without using viral vectors or any other materials subject to the NIH Guidelines and without integration of rsNA into the chromosome.

Genome 52

Genome Genomics Gene Editing Genetic Engineering 52

pharmaphorum

APRIL 20, 2022

The potential to develop treatments with CRISPR gene editing is hinged on its ability to add, delete or replace elements within a target DNA sequence. Cancer is a genetic disease, caused by certain changes in the way that genes control cell function, such as how they grow and divide. Why cancer? The pipeline.

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Gene Editing DNA Gene Genetics 52

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

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. They also offer guidance on clinical trial design and regulatory pathways to accelerate the approval of orphan drugs.

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Research Drugs Gene Therapy Clinical Trials 59

BioSpace

NOVEMBER 17, 2022

Editas Medicine is pausing its ocular gene therapy program after demonstrating a favorable safety profile and seeking a potential partner to develop EDIT-101, the company announced Thursday.

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Gene Therapy Gene Gene Editing Medicine 42

pharmaphorum

SEPTEMBER 14, 2022

SparingVision has raised €75 million in second-round funding that will be used to fund clinical trials of gene therapies for ocular diseases retinitis pigmentosa (RP) and dry age-related macular degeneration (AMD).

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Gene Therapy Gene Gene Sequencing Gene Editing 52

Bioengineer

JULY 23, 2021

“By applying high-resolution imaging, advanced cell and molecular readouts and RNA sequencing methods leveraging the OTME-Chip, we discovered the actual genetic signaling pathways behind the blood cell triggered metastasis of ovarian cancer and a new drug strategy to stop this process.” ” ###.

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Engineer Gene Editing Clinical Trials Clinical Trials 52

The Pharma Data

NOVEMBER 3, 2020

Nasdaq:LOGC) (LogicBio), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, announced today the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its clinical candidate, LB-001 for the treatment methylmalonic acidemia (MMA). LEXINGTON, Mass.,

Clinical Trials 40

Clinical Trials Clinical Trials Gene Editing Genome 40

Advarra

NOVEMBER 29, 2022

The BGTC will act as a traffic light for these therapies, providing information on basic and clinical research, manufacturing, production, and regulatory requirements. While each ultra-rare genetic disorder may have a very small patient population, when these conditions are grouped together, they make up a significant number.

Gene Therapy 52

Gene Therapy Gene Production Clinical Trials 52

pharmaphorum

JANUARY 29, 2021

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers. That would just slow the whole field down.

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Gene Editing Gene Gene Therapy In-Vivo 145

pharmaphorum

NOVEMBER 2, 2021

In 2019, there were 57 bsAb candidates in clinical trials, against both haematological and solid tumours. In the pipeline , Amgen has BiTE molecules targeting seven types of cancer in clinical trials, as well as further structures in the design stage. Engagement of immune cells to the tumour cell.

Antibody 52

Antibody Immune Response Production Engineer 52

Camargo

JULY 27, 2021

It is a comprehensive term which encompasses a large variety of therapy products including viral and bacterial vectors, plasmid DNA, human gene editing technology, and patient-specific cellular gene therapy. Additionally, gene editing allows us either to remove or to modify harmful genes.

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Gene Therapy Gene Gene Editing Genetics 238

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 69

Pharmaceutical Technology

AUGUST 24, 2022

A lead product candidate of the company, GTP-506 is a single-dose gene editing therapy. The ARCUS nuclease vector (GTP-506A) targets gene editing in the PCSK9 gene locus that is well characterised, while the therapeutic donor vector (GTP-506D) introduces the OTC gene to provide the required genetic correction.

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Gene Editing Gene Therapy Gene Genetics 130

STAT News

NOVEMBER 4, 2022

A man with muscular dystrophy who was first in line to receive an experimental gene editing therapy tailor made to treat the cause of his rare form of the disease has died. Terry had long been too old to participate in clinical trials of experimental therapies for the disease, which are often geared towards young boys.

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Gene Editing Gene Gene Therapy Scientist 105

pharmaphorum

AUGUST 8, 2022

The GBT deal comes at a time when the market for SCD therapies is undergoing significant change, with multiple new drugs reaching the market after years of stagnation and progress also being made with genetic therapies from the likes of bluebird bio, Vertex Pharma/CRISPR Therapeutics and Precision Bio/Novartis.

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Marketing Sales Gene Editing Antibody 111

The Pharma Data

JANUARY 10, 2021

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. The Company is required to develop and submit to the FDA an improved potency assay prior to enrolling the efficacy phase of the RUBY trial.

Medicine 52

Medicine Development Clinical Trials Clinical Trials 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. Additionally, Gag-specific CD8+ T cells demonstrated an increase of 1.7

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FDA Approval Gene Therapy Gene Clinical Trials 111

XTalks

DECEMBER 13, 2023

Casgevy’s approval by the FDA is momentous: it is the first CRISPR-based gene-editing therapy to be approved in the US. In 2022, bluebird won approvals for two gene therapies — Skysona for the treatment of the rare neurological disorder cerebral adrenoleukodystrophy (CALD) and Zynteglo for beta-thalassemia.

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Gene Therapy Gene FDA Approval In-Vivo 98

XTalks

MARCH 28, 2024

For CRC, treatment options that are targeting genetic mutations such as BRAF , KRAS and HER2 have been developed. Early clinical trials have shown promising results and this therapy could become a valuable addition towards CRC treatment in the future. Yang Liu and CFO Abid Ansari – Xtalks Life Science Podcast Ep.

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Gene Editing Life Science Clinical Trials Clinical Trials 59

XTalks

DECEMBER 29, 2022

In 2022 alone, the US regulator approved four new gene therapies, showing the high interest in getting these therapies to market. In 2023, a number of gene therapies are expected to get the FDA green light. Given their precise targeting, gene therapies have largely been focused on oncology indications followed by rare genetic diseases.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

The Pharma Data

NOVEMBER 16, 2020

16, 2020 — Scientists are reporting early success with an experimental herpes vaccine that uses a genetically modified version of the virus. The gene edit prevents the virus from performing its normal evasive maneuver: hiding out in nervous system cells in order to elude the immune system. MONDAY, Nov.

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Vaccination Vaccine Immune Response Genetics 52

XTalks

SEPTEMBER 4, 2024

Alnylam Pharmaceuticals announced promising results from its HELIOS-B Phase III clinical trial evaluating vutrisiran, an investigational RNA interference (RNAi) therapeutic for treating transthyretin amyloidosis with cardiomyopathy (ATTR-CM).

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Trials RNA Protein Gene Editing 52

The Pharma Data

OCTOBER 20, 2020

In hopes to advance the best and brightest, MD Anderson, in partnership with The Focus Fund GP, has launched the Cancer Focus Fund to propel compelling investigational cancer therapies from preclinical development through Phase II clinical trials. Targeted therapies, genetic sequencing, gene editing, immunotherapies.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Editing Trials 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 98

Worldwide Clinical Trials

MAY 30, 2024

Authors: Rich Worldwide Clinical Trials Exec. This trend is on the rise despite recent disappointments with clinical trial outcomes, which have the potential to destabilize the industry in the short term regarding drug development strategy and optimal study designs. Director, Therapeutic Area Medical Lead.

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Gene Clinical Trials Clinical Trials Gene Therapy 204

XTalks

DECEMBER 22, 2021

RNA therapeutics hold promise as powerful treatments for diseases, including those that are genetic-based. The gene-editing tool CRISPR edits DNA using specialized strands of guide RNA and endogenous cellular mechanisms of RNA interference. RNA Therapeutics. The deal is worth $1.5

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Life Science RNA Vaccination Vaccine 98

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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