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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Gene Therapy Gene Gene Editing Sales 111

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. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. Both therapies will be available in early 2024.

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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. .”

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Gene Editing Gene Gene Therapy Drugs 59

pharmaphorum

SEPTEMBER 28, 2022

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The post Vertex, CRISPR prep filing for gene-editing blood disorder therapy appeared first on.

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Gene Editing Gene In-Vivo Clinical Trials 59

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

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. The move marks a continuation in its ongoing commitment to exploring gene-editing technology.

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Gene Editing Gene Gene Therapy Genetics 52

Medical Xpress

FEBRUARY 7, 2023

A recent study by University of Illinois Chicago researchers in the College of Business Administration analyzed how the experiences of racism and discrimination in health care significantly affect the adoption of innovative medical technology like gene therapies and the gene editing tool CRISPR.

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Gene Editing Gene Therapy Gene Marketing 71

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. Common gene therapies use vehicles such as adeno-associated vectors (AAVs), which cannot carry the required payload in this case, says Carney.

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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

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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Gene Editing Gene In-Vivo Genetics 52

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

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., “Accessing state-of-the-art LNP technology through this collaboration will add momentum to our gene editing efforts for the benefit of patients.”

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Gene Therapy Gene Gene Editing In-Vivo 40

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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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.

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Life Science Gene Therapy Gene Editing Gene 98

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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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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Life Science Gene Editing Development Clinical Trials 119

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. greater safety, transient and reversible effect on gene expression).

Genetics 52

Genetics Medicine Gene Therapy Marketing 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 In-Vivo 40

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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In-Vivo Gene Editing Trials Gene 52

pharmaphorum

FEBRUARY 17, 2022

Any near-term impact in Europe will be limited after Bluebird Bio pulled its Zynteglo (betibeglogene autotemcel) gene therapy for another rare blood disorder, beta thalassaemia, off the market in Germany after failing to secure agreement on pricing.

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Drugs Gene Therapy Gene Editing Gene 59

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

Gene and cell therapies hold the potential to provide a one-off cure. The major drivers that have enabled this vision are the advent of safe and effective vectors, newer gene-editing tools, and huge unmet needs for the treatment of devastating CNS disorders. Competition in the Market. Race to be the first mover.

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Gene Gene Therapy Gene Editing Development 52

Pharmaceutical Technology

MAY 26, 2023

Tripling its production capacity, Touchlight can now manufacture 8kg plasmid DNA, a key component for mRNA gene therapies and vaccines. Research into genetic medicines like mRNA vaccines and gene therapies, as well as gene-editing treatments and novel cancer therapies, have driven increased demand for DNA.

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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.

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Life Science Gene Therapy Gene Manufacturing 52

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. In addition, safety oncology and immunotherapy, which is a focus area for Cerba Research has close ties to virology.

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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.

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Bioassay Gene Editing Marketing Gene Therapy 52

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. Both therapies were awarded their approvals on the same day in December 2023.

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XTalks

SEPTEMBER 20, 2024

Chimeric antigen receptor T-cell therapy (CAR T) has provided substantial benefit to patients with certain hematological malignancies and has significant opportunity for growth. As of 2024, the global CAR T-cell therapy market is valued at $4.6 billion and is projected to reach $15.2 billion by 2035.

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Gene Editing Manufacturing Gene In-Vivo 52

Cloudbyz

MARCH 6, 2024

As we continue to unlock the mysteries of the human body and harness the power of biotechnology, the era of “beyond the pill” therapies is well underway, promising a future where diseases are not just managed, but truly conquered.

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Gene Immune Response Manufacturing Gene Therapy 99

The Pharma Data

MARCH 11, 2021

Speaking at the virtual Capital Markets Day on Wednesday, CEO Werner Baumann emphasized Bayer’s long-term growth perspectives: “Bayer is a leading life science company, uniquely positioned at the intersection of health and nutrition – with attractive prospects for growth, earnings and cash flow.” In addition to further strengthening its U.S.

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Sales Gene Therapy Marketing Gene 52

The Pharma Data

DECEMBER 28, 2020

Scopus is a biopharmaceutical company whose lead drug candidate is a novel, targeted immuno-oncology gene therapy for the treatment of multiple cancers. Mr. Weild is a globally recognized leader in capital formation and capital markets structure. ” The new directors are: Raphael (“Rafi”) Hofstein, Ph.D.

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Gene Therapy Life Science Gene Gene Editing 52

Pharma Marketing Network

JANUARY 20, 2021

In their annual Cell & Gene Therapy State of the Industry Briefing, the Alliance for Regenerative Medicine identified as many as 1085 gene and cell therapy companies worldwide.

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Vaccination Vaccine In-Vivo Genetics 52

The Pharma Data

NOVEMBER 3, 2020

In addition, LogicBio has a collaboration with Takeda to research and develop LB-301, an investigational therapy leveraging GeneRide for the treatment of the rare pediatric disease Crigler-Najjar syndrome. LogicBio is also developing a Next Generation Capsid platform for use in gene editing and gene therapies.

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Clinical Trials Clinical Trials Gene Editing Genome 40

XTalks

AUGUST 2, 2023

Join us as we present an in-depth analysis of each company’s revenue, net income, R&D investments, core therapeutic areas, market presence and strategic collaborations. is a global pharmaceutical company, working across both developed and emerging markets. 2022 Revenue: Pfizer reported an annual revenue of $100.33 Pfizer Inc.

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XTalks

DECEMBER 24, 2020

From rare disease drug approvals to treatments involving immunotherapies and gene therapies and awarding of a Nobel Prize to the inventors of the gene-editing tool CRISPR, 2020 was a year of great activity and productivity despite the backdrop of the pandemic. CRISPR Gene Editing Inventors Win Nobel Prize.

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XTalks

JANUARY 6, 2025

While these therapies span all stages of clinical development, they are particularly concentrated in earlier phases, indicating strong future growth. RNA-based therapies hold the potential to offer new treatment options for diseases with limited or inadequate therapeutic alternatives. The global stem cell market was valued at $15.07

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