pharmaphorum

OCTOBER 4, 2022

AstraZeneca’s rare disease firm Alexion is set to expand its genomic medicine portfolio with the acquisition of gene editing specialist LogicBio Therapeutics, in a deal worth approximately $68 million. The post AstraZeneca pays record 660% premium for gene editing company LogicBio appeared first on.

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pharmaphorum

AUGUST 24, 2021

Vertex Pharma has ramped up its involvement in gene-editing medicines for the third time in a matter of months, agreeing a partnership with CRISPR specialist Arbor Biotechnologies that could be worth up to $1.2 If approved, it could mount a challenge to bluebird bio’s gene therapy Zynteglo, which is already approved in Europe.

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pharmaphorum

OCTOBER 13, 2022

Gene therapies and research into them have grown immensely in recent years, offering more novel tools in regenerative medicine to fight disease, including rare diseases and genetic disorders. Beta-thalassemia is a rare blood disorder caused by a genetic defect in hemoglobin. DOWNLOAD THE FULL ARTICLE HERE.

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Pharmaceutical Technology

FEBRUARY 13, 2023

Gene therapies are an attractive way of targeting the underlying genetic mutations, but traditional approaches cannot be used while targeting mutations for Dravet Syndrome, forcing researchers to develop new ways. Meanwhile, others are researching the link between gene variation and different responses to treatments.

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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). billion in potential milestones if the project advances through development and onto the market. billion agreement that started in 2020.

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pharmaphorum

AUGUST 8, 2022

As we reported this morning, the deal gives Pfizer already-approved SCD therapy Oxbryta (voxelator) – which industry watchers reckon could see a dramatic uptick in sales with Pfizer’s marketing muscle – plus a phase 3 antibody candidate, a phase 1 follow-up to Oxbryta that could offer improved dosing.

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pharmaphorum

APRIL 23, 2021

Vertex Pharma has ramped up its involvement in gene-editing medicines for the second time in a week, paying Obsidian Therapeutics $75 million upfront to access its technology platform. . Alternatively, a drug can be administered that switches off the genetic medicine and brings protein expression to a halt.

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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.

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pharmaphorum

APRIL 23, 2021

CRISPR Therapeutics is to receive a hefty $900m payment from Vertex after the companies amended a collaboration to develop, manufacture and market a gene editing therapy for sickle cell disease and beta thalassemia.

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BioPharma Reporter

AUGUST 30, 2021

Boston-based company, Vertex, has signed a deal to use Arbor Biotechnologiesâ CRISPR gene-editing technology to develop novel cell therapies for the treatment of serious diseases.

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XTalks

DECEMBER 20, 2023

CRISPR Gene Editing Therapeutics CRISPR/Cas-9 technology makes lists like this almost every year, and for good reason. Given its ability to precisely edit genes, there is tremendous promise for the revolutionary technology to offer cures for genetic disorders and to treat various diseases.

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pharmaphorum

MAY 7, 2021

Gene editing firm CRISPR Therapeutics has announced a collaboration with US biotech Nkarta to develop natural killer (NK) cell therapies for cancer. It’s at the forefront of gene editing although the technology has spawned rivals including Intellia Therapeutics, Caribou Biosciences, Sangamo Therapeutics and Mammoth Biosciences.

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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.

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Pharmaceutical Technology

MARCH 2, 2023

The research using ImmunoGen’s technology will enable the discovery of the agents for use with gene editing. ImmunoGen has announced an agreement granting rights to Vertex Pharmaceuticals to carry out research using antibody-drug conjugate (ADC) technology for the discovery of new targeted conditioning agents.

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Worldwide Clinical Trials

MAY 30, 2024

Since marketing authorization for the first breakthrough treatment in 1994, the steady increase in clinical trials reflects the community’s commitment to finding effective ALS treatments despite the numerous hurdles associated with clinical trial design, from proof-of-concept to pivotal trials. Director, Therapeutic Area Medical Lead.

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Roots Analysis

FEBRUARY 15, 2022

Presently, there are several companies and universities, which are exploring the potential of different gene editing technologies beyond CRISPR for basic research, and the development of gene editing solutions. There are various genome editing technologies which use enzymes that recognize and attach on to specific sites.

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XTalks

MAY 22, 2023

Related: 5 Food Companies Working With Precision Fermentation Technology How Pairwise Leverages CRISPR Technology CRISPR, or Clustered Regularly Interspaced Short Palindromic Repeats, is a gene-editing technique that can be used to alter the DNA of cells to enhance certain characteristics or reduce less desirable ones.

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pharmaphorum

AUGUST 31, 2022

After a recent approval, there are now three gene therapies available on the US market. Though there are still few commercial treatments, in terms of therapeutic potential, Ben Hargreaves finds that companies and investment firms are pushing for this number, and the overall market, to grow rapidly in the coming years.

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XTalks

JANUARY 4, 2024

Hemophilia B, also known as congenital Factor IX (FIX) deficiency or Christmas disease, is a rare genetic bleeding disorder caused by a deficiency or defect in the FIX clotting protein. Having an early presence in the market is particularly beneficial for gene therapies, which are often designed as single-dose treatments.

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pharmaphorum

FEBRUARY 17, 2022

SCD is one of the most prevalent genetic diseases in Europe, affecting approximately 52,000 people, and is more common in those whose ancestors are from sub-Saharan Africa, although it is also seen in people of Hispanic, South Asian, Southern European and Middle Eastern ancestry.

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pharmaphorum

NOVEMBER 1, 2022

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. Director, Genetic Medicine. Website: [link].

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pharmaphorum

JANUARY 10, 2022

The first mRNA-based vaccines were brought onto the market in 2020, with Pfizer/BioNtech and Moderna receiving emergency use authorisation from the US Food and Drug Administration (FDA) for the prevention of COVID-19. The breakthrough was hailed as a revolution for disease treatment and particularly a major success against the pandemic.

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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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Delveinsight

JANUARY 14, 2021

The multi-arm trial will gauge various combinations of selgantolimod, Gilead’s investigational TLR-8 agonist; VIR-2218, Vir’s investigational small interfering ribonucleic acid (siRNA); and a commercially-sourced, marketed PD-1 antagonist. Verve Therapeutics unveils its lead program for treating genetically high cholesterol.

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Pharmaceutical Technology

FEBRUARY 20, 2023

Gene therapy, while in its infancy, could be a gamechanger for cancer treatment and certain rare diseases and advancements in gene-editing technology relies on knowledge of viral factors. The technology platforms are almost the same. The opportunity here for our expanded CRO to advance personalised medical solutions is huge.

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Cloudbyz

MARCH 6, 2024

Personalized Medicine: Biologics offer the potential for personalized treatment approaches by taking into account individual variations in genetics, immune responses, and disease characteristics. Biosimilars have the potential to lower healthcare costs, increase market competition, and improve patient access to biologic therapies.

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pharmaphorum

OCTOBER 8, 2021

The company is also seeking if there is evidence of clonal expansion of the abnormality in in other words passed on to daughter cells – and if it is linked to the gene-editing used to modify the ALLO-501A cells.

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Pharmaceutical Technology

MAY 26, 2023

As per the company, dbDNA is suitable for rapid, scalable manufacture of GMP DNA and can incorporate gene sequences of sizes ranging from 500bp to 20kb, which are typically unstable as pDNA. Touchlight estimates that the DNA market is currently worth $1bn and will grow 20% annually until 2030.

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The Pharma Data

JANUARY 17, 2021

–( BUSINESS WIRE )– Genus plc (LSE: GNS), a global biotechnology leader in animal genetics, today reported continued strong performance across its operations for the six-months period ending December 31, 2020. 18, 2021 07:00 UTC. BASINGSTOKE, England & MADISON, Wis.–( and £49.0m

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pharmaphorum

SEPTEMBER 14, 2022

The French biotech said the cash injection – which follows a €45 million Series A in 2020 – will also help it bring forward the first development candidate in a collaboration signed with gene-editing specialist Intellia Therapeutics last year, currently in the early-discovery stage.

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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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The Pharma Data

OCTOBER 20, 2020

This is where biotechs are essential to take promising discoveries from the research lab into a product that can be tested, approved and taken to market. Targeted therapies, genetic sequencing, gene editing, immunotherapies.

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The Pharma Data

JANUARY 10, 2021

EDIT-301 is an experimental, ex vivo gene editing cell medicine in development for the treatment of sickle cell disease. Previously, the Company received Rare Pediatric Disease designation from the FDA for EDIT-301. EDIT-301 is the first experimental medicine in development generated using CRISPR/Cas12a gene editing.

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XTalks

DECEMBER 22, 2021

The market for mRNA-based vaccines and therapeutics is projected to grow from $46.7 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.

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Pharma Marketing Network

JANUARY 20, 2021

In other areas of the industry (such as cell and genetic medicine development) progress continued at an impressively brisk pace despite the pandemic. The field of genetic medicine is continuing to make great advances, despite the challenges of the pandemic.

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XTalks

DECEMBER 29, 2022

This highlights potentially new marketing trends that life science companies may have to start looking into if they haven’t already. In 2022 alone, the US regulator approved four new gene therapies, showing the high interest in getting these therapies to market. 4. Social Media Marketing.

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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. These regulations offer benefits such as tax credits, grant funding and extended market exclusivity for approved drugs.

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