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. billion deal with Precision Biosciences to support its development of one-time treatments for beta-thalassemia and sickle cell.

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Gene Editing Gene Gene Therapy Genetics 52

pharmaphorum

JUNE 12, 2022

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has continued to show impressive results in clinical trial, with an update at the EHA congress showing long-term effects on the symptoms of both beta thalassaemia and sickle cell disease. billion product if it gets approved for both indications. .”

Gene Editing 57

Gene Editing Gene Gene Therapy Drugs 57

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

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.

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Gene Editing DNA Gene Gene Therapy 235

Drug Discovery World

SEPTEMBER 6, 2022

In recent years the advanced therapy industry has been the most significant disruptor in medicine. Cell and gene therapies are not only transforming the way diseases are treated but are also aiming to cure entirely new patient populations. ViaCyte, Beam, and Verve are all due to begin gene editing trials this year.

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Gene Therapy Gene Gene Editing Manufacturing 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

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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Gene Editing Gene Genome Genomics 62

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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Gene Therapy Gene Gene Editing In-Vivo 40

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

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.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

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. We’re seeing a drive towards the allogeneic market with more progressing into clinical trials.

Gene Therapy 52

Gene Therapy Gene Gene Editing Production 52

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.

Genome 40

Genome Genomics Gene Gene Editing 40

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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Gene Therapy Gene Antibody FDA Approval 59

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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Gene Editing In-Vivo Gene Sequencing Gene 81

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.

Development 98

Development Drugs Clinical Trials Clinical Trials 98

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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Marketing Sales Gene Editing Antibody 103

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.

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In-Vivo Gene Editing Trials Gene 52

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.

Drugs 59

Drugs Life Science Gene Editing Gene 59

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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Gene Clinical Trials Clinical Trials Gene Therapy 163

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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Life Science Gene Editing Development Clinical Trials 119

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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Research Gene Gene Therapy Genome 264

XTalks

FEBRUARY 27, 2024

Technologies like CRISPR-Cas9 have revolutionized the potential for gene editing, offering hope for curative treatments for conditions like Duchenne muscular dystrophy (DMD) and certain types of inherited blindness. These regulations offer benefits such as tax credits, grant funding and extended market exclusivity for approved drugs.

Research 59

Research Drugs Gene Therapy Clinical Trials 59

pharmaphorum

DECEMBER 14, 2022

Healthcare marketing agency Klick Health is going global, and with that come several new hires: global managing directors Jennifer Lambert and Roberta Raduan and operations professionals Michael Becker , Shaun Chalk , and Catherine MacInnis. Boyds hires cell and gene therapy expert. Bevy of hires at Klick Health.

Life Science 97

Life Science Gene Therapy Gene Editing Gene 97

Pharma Marketing Network

DECEMBER 21, 2020

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. Here are 5 considerations when developing a communications strategy for a novel genetic medicine. #1.

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

Drug Discovery World

DECEMBER 20, 2022

As 2022 draws to a close, life sciences market analysts attempt to correctly predict what the next 12 months might bring to the drug discovery sector. DDW’s Diana Spencer summarises the key trends likely to impact the market in 2023. . There will be greater emphasis on diversity in clinical trials. AI will take on a wider role.

Drugs 52

Drugs Gene Editing Clinical Trials Clinical Trials 52

Drug Discovery World

APRIL 15, 2024

Gene therapy will move to ex vivo gene modification and the delivery of gene-modified cell types.

Gene Editing 59

Gene Editing Clinical Trials Clinical Trials In-Vivo 59

XTalks

DECEMBER 29, 2022

For example, on the pharma and biotech side, there has been growing demand for new drugs and biologics, from new classes of dual-action diabetes and weight loss drugs like Ozempic and Mounjaro to life-saving gene therapies. 1. Cell and Gene Therapies. Here are four life sciences trends to look out for in 2023.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

Drug Discovery World

JULY 10, 2023

If there’s one thing I would take from the Summer 2023 issue of Drug Discovery World , it would be that the application of technologies and processes throughout the pharmaceutical industry are changing how scientists pursue the development of therapies. There are myriad ways that this is being enabled.

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Gene Therapy Gene Scientist Gene Editing 52

pharmaphorum

SEPTEMBER 29, 2020

As of 2018, there were over 1,100 cancer therapies in development, and as of 2020, 362 of them were cell and gene therapies. The industry is asking itself how to stay innovative, how to develop and bring to market higher quality therapies to patients – and how to do this faster and more efficiently.

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Research Gene Editing Gene Development 117

Drug Discovery World

APRIL 3, 2023

I quickly recognised the commercial value of aptamers as being able to address the gap in the market where antibodies fail to perform, and together we established the business to begin aptamer development. Addressing this need in the market has allowed us penetration to work with 75% of the top twenty pharma companies.

Antibody 52

Antibody Gene Therapy Gene In-Vitro 52

The Pharma Data

JANUARY 17, 2021

18, 2021 /PRNewswire/ — Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Lonza announced today their agreement in support of ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD). NEW YORK , Jan.

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Manufacturing Gene Therapy Trials Gene 52

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.

Gene 52

Gene Gene Therapy Gene Editing Development 52

Roots Analysis

JANUARY 16, 2023

At-home Self-testing Kit Manufacturers – Current Market Landscape. The market is highly fragmented, featuring the presence of both new entrants and established players, based in different geographical regions. At-home Self-testing Kits – Market Overview. At-home Self-testing Kits – Market Forecast and Opportunity Analysis.

Bioassay 52

Bioassay Gene Editing Marketing Gene Therapy 52

Drug Discovery World

SEPTEMBER 6, 2022

The cell and gene therapy industry has experienced exponential growth in the last three years and as funding for the field continues to break records there are no signs of a slowdown. With 2,261 ongoing clinical trials in regenerative medicine, the FDA expects to approve 1 between 10 and 20 new cell and gene therapies a year by 2025.

Gene Therapy 52

Gene Therapy Gene Manufacturing Clinical Trials 52

Drug Discovery World

OCTOBER 16, 2023

Alchemab, on the other hand, has developed a highly differentiated platform which enables the identification of novel drug targets and therapeutics by analysis of patient antibody repertoires, drawing from the expertise in Cambridge to progress the company’s mission to build a broad pipeline of protective therapeutics for hard-to-treat diseases.

Drugs 75

Drugs Life Science Gene Therapy Genome 75

Cloudbyz

MARCH 6, 2024

The Evolution of Pharmaceutical Therapies: The emergence of biologics represents a significant paradigm shift in the pharmaceutical industry, prompting a reevaluation of traditional drug development and manufacturing processes. Challenges and Future Directions: Despite their immense promise, biologics are not without challenges.

Gene 73

Gene Immune Response Manufacturing Gene Therapy 73

Drug Discovery World

JANUARY 9, 2024

Thirteen new-to-market therapeutics and drugs poised to launch in 2024 will achieve ‘blockbuster’ status by 2029 or deliver game-changing benefits to patients. The therapy has shown positive results in Phase III trials in both breast and lung cancer.

Drugs 69

Drugs Gene Editing Antibody Medicine 69