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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Drug Discovery World

MAY 2, 2024

Hailed as a revolution in the treatment of many diseases, cell and gene therapy (CGT) is the fastest growing area of therapeutics. Perhaps most significantly, Casgevy (or exa-cel) from Vertex Pharmaceuticals and CRISPR Therapeutics, was approved to treat sickle cell disease and became the first gene therapy that uses CRISPR gene editing.

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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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Pharmaceutical Technology

MARCH 28, 2023

Vertex Pharmaceuticals has signed a new non-exclusive licensing agreement with CRISPR Therapeutics to expedite the development of its hypoimmune cell therapies to treat type 1 diabetes (T1D). The gene-editing technology allows for precise, directed changes to genomic DNA. The system comprises the Cas9 enzyme and a guide RNA.

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Roots Analysis

FEBRUARY 27, 2024

The global genome editing market is anticipated to grow at a CAGR of 12.6% How is the genome editing market landscape evolving: Currently, there is an evident increase in demand for complex biological therapies (including regenerative medicine products), which has created an urgent need for robust genome editing techniques.

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BioPharma Reporter

MARCH 28, 2023

Vertex will pay CRISPR Therapeutics $100m up-front for non-exclusive rights to its technology with the goal of developing a certain type of therapy for diabetes.

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Drug Discovery World

OCTOBER 24, 2023

While attending Life Science Baltics 2023, Diana Spencer spoke to Monika Paule , CEO and Co-founder of Caszyme, about creating a company based on CRISPR gene editing and some of the challenges of operating in this dynamic area of research. Caszyme was founded in 2017 by the pioneers of CRISPR gene editing technologies.

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XTalks

JANUARY 4, 2024

Pfizer has kickstarted the new year with its first-ever gene therapy approval, awarded by Health Canada to the company’s Beqvez (fidanacogene elaparvovec) for the treatment of hemophilia B. There is a significant focus on developing gene therapies as longer-term solutions for the disease.

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Scienmag

NOVEMBER 11, 2021

Sprouted barley fetches considerably low market prices and poses an economic burden on farmers and corporations that are at the mercy of nature to survive in the agriculture industry. After a spell of unexpected rain, before the harvest season, a farmer may be faced with the unpredictable problem of untimely sprouting of barley.

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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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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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XTalks

OCTOBER 20, 2023

Intellia said NTLA-2001 is the first investigational in vivo CRISPR-based gene editing therapy cleared to enter late-stage clinical development. As an in vivo therapy, it can edit genes inside the body rather than in cells extracted from patients. ATTR amyloidosis is a rare, progressive and fatal disease.

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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., a biotechnology company specializing in the development of lipid nanoparticle (LNP) delivery systems for molecular therapeutics.

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Drug Discovery World

SEPTEMBER 6, 2022

Cell and gene therapies are not only transforming the way diseases are treated but are also aiming to cure entirely new patient populations. As researchers and developers work to transform the way we treat diseases, cell and gene therapies will continue to be the centre of innovation in the coming years. . billion raised.

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XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel).

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Gene Therapy FDA Approval Gene In-Vivo 98

Drug Discovery World

JUNE 29, 2023

This 24-page eBook sponsored by The Jackson Laboratory looks at the key use-cases for genomics and how it is improving drug discovery and development projects.

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Genome Genomics Gene Editing Drugs 52

Drug Discovery World

DECEMBER 11, 2023

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is also the first FDA-approved treatment to use CRISPR gene editing technology.

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Gene Therapy FDA Approval Gene Gene Editing 52

Drug Discovery World

JUNE 29, 2023

This 24-page eBook sponsored by The Jackson Laboratory looks at the key use-cases for genomics and how it is improving drug discovery and development projects.

Genome 52

Genome Genomics Gene Editing Drugs 52

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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Drug Discovery World

AUGUST 11, 2023

GenScript Biotech and T-MAXIMUM Biotech have formed a strategic collaboration to develop T-MAXIMUM’s CAR-T cell therapy using GenScript’s CRISPR nucleic acid reagents. GenScript will provide various CRISPR reagents to support the development of the universal CAR-T products from discovery to commercialisation.

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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). The post SparingVision raises €75m for eye disease gene therapies appeared first on.

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Gene Therapy Gene Gene Sequencing Gene Editing 52

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.

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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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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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Drug Discovery World

MAY 23, 2023

DDW’s Megan Thomas caught up with George White , General Manager, Product Management, Cell & Gene Therapy at Cytiva, to learn more about the company’s insight into the CGT market. Key challenges for cell and gene therapies In order for cell and gene (CGT) therapies to succeed, George White identifies several challenges to overcome.

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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

DECEMBER 20, 2023

As we step into 2024, the life sciences continue to evolve at an unprecedented pace, driven by technological innovation, a deeper understanding of human biology and the application of new technologies in areas like drug development and health wearables. Regulatory bodies are also taking note of the applications of AI in drug development.

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Drug Discovery World

JANUARY 26, 2023

DDW Editor Reece Armstrong takes a look at the pharmaceutical research and development market in 2023 and asks key opinion leaders what challenges and trends will emerge throughout the year. billion showed the pharma giant taking further steps into the oncology market and developing its speciality medicines vaccines portfolio.

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Drug Discovery World

MAY 6, 2024

In this article, DDW’s Diana Spencer highlights several prominent hotspots that are taking the lead on advancing European drug discovery and development. During the period 2016-2021, the Brazilian, Chinese and Indian markets grew by 11.7%, 6.7% respectively compared to an average market growth of 5.8% for the US market 2.

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Drugs Life Science Gene Editing Gene 59

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. . Vertex is taking over 60% of the costs of the programme in return for getting the same proportion of profits on sales if CTX001 reaches the market.

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Gene Editing Genetics Drugs Gene 52

Drug Discovery World

MAY 9, 2024

Mekonos, a company working towards cell engineering on a chip, has entered into a partnership agreement with Accelerated Bio to codevelop a new manufacturing cell line based on Accelerated Bio’s human Trophoblast Stem Cells (hTSCs) and Gene Engineered Multi-Sites (GEMS) technology.

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Engineer Gene Editing Manufacturing Gene 64

Pharmaceutical Technology

FEBRUARY 13, 2023

Several biotech companies and researchers are now exploring medical devices and gene therapies to address not just common forms of epilepsy, but also rare conditions such as Dravet Syndrome. Meanwhile, others are researching the link between gene variation and different responses to treatments.

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Drug Discovery World

DECEMBER 21, 2023

DDW’s top 10 most popular articles for 2023 reflect the key subjects in drug discovery and development. In a record year for gene therapy approvals, it isn’t surprising that cell & gene therapy (CGT) has been a hot topic. DDW Multimedia Editor Megan Thomas explored three trends in the market.

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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. Genome Editing is a way of making changes in the DNA. Meganucleases. Our Social Media Platform.

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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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Delveinsight

FEBRUARY 25, 2021

For most CNS disorders, the development of effective drug candidates is challenging as clinical trials are often long-lasting and with considerable expense. The last few decades have been marked by increased research and development in neuroscience. Positioning of Gene and Cell Therapies in Neurosciences. Early Victories.

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Pharmaceutical Technology

MARCH 1, 2023

Nedisertib is under clinical development by Merck and currently in Phase I for Neuroendocrine Tumors. GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Merck overview Merck, a subsidiary of E.

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