pharmaphorum

JULY 9, 2021

Orchard Therapeutics’ gene therapy for rare childhood disease metachromatic leukodystrophy (MLD) Libmeldy has been rejected for NHS use by NICE in draft guidance. The post NICE rejects Orchard’s gene therapy for rare childhood disease MLD appeared first on.

Gene Therapy 105

Gene Therapy Gene In-Vivo Marketing 105

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 97

XTalks

AUGUST 19, 2022

Bluebird bio’s gene therapy Zynteglo (betibeglogene autotemcel, beti-cel) has been awarded a much anticipated approval from the US Food and Drug Administration (FDA) for the treatment of adult and pediatric patients with beta thalassemia who need regular blood transfusions. Bluebird has a total of three gene therapies in its pipeline.

Gene Therapy 95

Gene Therapy FDA Approval Gene Clinical Trials 95

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.

In-Vivo 52

In-Vivo Gene Editing Trials Gene 52

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. Casgevy is also the first ever CRISPR/Cas9-based therapy approved in the US.

Gene Therapy 98

Gene Therapy Gene FDA Approval In-Vivo 98

XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

World of DTC Marketing

JANUARY 31, 2021

Smaller biotechs that are developing new drugs usually don’t have the resources to take new drugs to market. The cost of bringing a drug to market decreased from $2,168 million in 2018 to $1,981 in 2019. Still, she is missing a lot of facts at a time when hospital and health insurance costs are soaring. percent from 2018.

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In-Vivo Generic Drugs Drugs Gene Therapy 187

Pharmaceutical Technology

AUGUST 17, 2022

Merck (MSD outside North America) has entered a partnership agreement with Orna Therapeutics for discovering, developing and marketing various programmes based on next-generation RNA technology. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

RNA 147

RNA Vaccine Vaccination In-Vivo 147

Pharmaceutical Technology

SEPTEMBER 14, 2022

As an autologous cell therapy, manufacturing the infusion product is a highly specialised process involving tumour harvest and the extraction and large-scale ex vivo expansion of TILs. Tumour-infiltrating lymphocyte (TIL) therapy has been investigated as a treatment modality for melanoma for almost 20 years. versus 21.4% respectively.

Immune Response 246

Immune Response In-Vivo Production Manufacturing 246

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. dosing of *first patient* with VERVE-101, an in vivo CRISPR base editing medicine. Today: we are announcing.

Gene Editing 98

Gene Editing Trials Drugs In-Vivo 98

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 time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market.

Gene Editing 59

Gene Editing Gene In-Vivo Clinical Trials 59

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 59

Gene Editing Gene Gene Therapy Drugs 59

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.

Genomics 40

Genomics Genome Gene In-Vivo 40

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

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

Pharmaceutical Technology

MARCH 9, 2023

Innovation S-curve for the pharmaceutical industry Transgenic murine models is a key innovation area in pharmaceutical s Transgenic murine models refer to mice that have been genetically altered for the purposes of understanding the in vivo functions of genes.

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In-Vivo Antibody Genetics Gene 130

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.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

The Pharma Data

JANUARY 17, 2021

But that number doesn’t quite reflect the company’s likely market valuation , which is expected to be between $9 billion and $12 billion. Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases.

In-Vivo 52

In-Vivo Marketing Engineer Genetics 52

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. Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e.,

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

pharmaphorum

OCTOBER 8, 2021

This year, we’ll be specifically focusing on next generation cell therapies and discussing: Novel in vivo delivery. This year, we’ll be specifically focusing on next generation cell therapies and discussing: Novel in vivo delivery. next generation innovations in cell therapies.

In-Vivo 52

In-Vivo Gene Manufacturing Marketing 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.”

Gene Therapy 40

Gene Therapy Gene Gene Editing In-Vivo 40

Delveinsight

JANUARY 18, 2021

Angelman syndrome is a result of the missing or dysfunctional gene known as UBE3A. The gene is responsible for the normal and healthy development of an individual physically as well intellectually. The present Angelman syndrome treatment market revolves around the management of symptoms rather than curing the condition. .

Marketing 52

Marketing Gene Drugs Protein 52

Roots Analysis

JANUARY 27, 2025

The American Society of Gene and Cell Therapy (ASGCT) defines cell therapy as a therapeutic modality that involves the administration of either normal or modified cells to patients for the treatment of various diseases. The major steps involved in cell therapy manufacturing have been briefly outlined below.

Manufacturing 40

Manufacturing Contamination Production Gene Therapy 40

pharmaphorum

MAY 27, 2021

Life sciences tools company Esco Lifesciences Group has scored $200m in a series A and crossover round co-led by Novo Holdings and Vivo Capital. Funds will be used to expand the company’s presence across Asia and create an innovation hub in Boston for cell and gene therapy technologies.

Life Science 59

Life Science In-Vivo Manufacturing Gene Therapy 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.

Gene Editing 52

Gene Editing Genetics Drugs Gene 52

The Pharma Data

NOVEMBER 10, 2020

This model has the benefit of giving companies direct oversight of their in vivo studies, allowing them to move faster while operationalizing every dollar of their seed financing. Photo courtesy of Mispro. There’s an emerging business model in biotech town, and it’s called the Contract Vivarium (CV).

Marketing 52

Marketing In-Vivo Scientist Research 52

Roots Analysis

MARCH 1, 2023

Disruption of the integrity and function of DNA at the gene or chromosomal level can lead to heritable mutations, resulting in genetic disorders, birth abnormalities, or cancer. Mechanism of Genotoxicity / Mutagenicity The interaction of genotoxins / mutagens with the structure of DNA causes damage to the genetic material.

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Genotoxicity DNA Genetics Bacteria 52

The Pharma Data

AUGUST 3, 2021

SVP, Chief Medical Officer, Novartis Gene Therapies. “We SVP, Chief Medical Officer, Novartis Gene Therapies. “We Additionally, STEER will add to the clinical data and emerging real-world evidence for the use of gene therapy to treat SMA. 2 years and <5 years old with SMA Type 2.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Gene 52

XTalks

SEPTEMBER 20, 2024

As of 2024, the global CAR T-cell therapy market is valued at $4.6 Vittoria Biotherapeutics is a clinical-stage cell therapy company that incorporates the latest advancements in cell therapy and gene editing with a unique approach that potentially overcomes some of the issues with this therapeutic modality. billion by 2035.

Gene Editing 52

Gene Editing Manufacturing Gene In-Vivo 52

The Pharma Data

NOVEMBER 22, 2020

Dr Squinto is currently Executive Partner of the healthcare investment company OrbiMed Advisors and has more than 25 years’ experience in the biotech industry, including as Chief Executive Officer of the gene therapy company Passage Bio. MILAN, Italy and NEW YORK, Nov. About Genenta Science.

Gene Therapy 40

Gene Therapy Gene In-Vivo Clinical Trials 40

Delveinsight

JANUARY 12, 2021

The companies believe that their candidate in its original IgG format has shown potent neutralization activity in in vitro assays and in an in vivo animal model. Already in markets in China, BeiGene’s Tislelizumab is a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to Fc? and Europe.

Licensing 52

Licensing Licensing Antibody Gene Therapy 52

The Pharma Data

OCTOBER 15, 2020

Axovant – Kristin Vuori was named to the board of directors at Axovant Gene Therapies Ltd. marketing and diagnostics. Assembly Biosciences – Former Gilead Sciences CEO John McHutchison, currently president and CEO of Assembly, was named the recipient of the Advance 2020 Global Impact award. BeyondSpring – BeyondSpring Inc.

In-Vivo 52

In-Vivo Business Development Manufacturing Development 52

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. Regarding OWS’s support for vaccine development, Dr Slaoui noted that 5 of the 6 vaccines selected (from a total of 94 programs) are currently in phase 3 development or approved.

Vaccine 52

Vaccine Vaccination In-Vivo Genetics 52

Roots Analysis

SEPTEMBER 18, 2023

Owing to the ability to preserve a wide variety of elements for such a long period of time, including cells, tissues, blood and DNA, cryopreservation has emerged as a key focus area for stakeholders in the biopharmaceutical industry. Therefore, in order to store and preserve such enormous amounts of blood, single-use assemblies are a necessity.

Engineer 52

Engineer Containment In-Vivo Production 52

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. EDIT-301 is the first experimental medicine in development generated using CRISPR/Cas12a gene editing. Previously, the Company received Rare Pediatric Disease designation from the FDA for EDIT-301.

Medicine 52

Medicine Development Clinical Trials Clinical Trials 52

The Pharma Data

DECEMBER 27, 2020

PARIS–( BUSINESS WIRE )– Regulatory News: Lysogene (FR0013233475 – LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today reports positive biomarker data from the ongoing AAVance clinical trial with LYS-SAF302 for the treatment of MPS IIIA (NCT03612869). About Lysogene.

Gene Therapy 40

Gene Therapy Clinical Trials Clinical Trials In-Vivo 40

Roots Analysis

JANUARY 14, 2024

Usually, the desired gene, such as human insulin gene, when inserted into the plasmid of the host cell uses transcriptional and translational machinery of the host to express itself. It is worth mentioning that in vitro gene expression requires a suitable host for the production of a specific gene product.

Contract Manufacturing 40

Contract Manufacturing Protein Manufacturing Production 40

Roots Analysis

AUGUST 23, 2024

This alone makes them much safer than most conventional vaccines on the market today. In recent years, mRNA technology has rocked the world of medicine, making it possible to prevent and treat a vast array of diseases and disorders. This modern marvel wasn’t just used against COVID-19 but can be tailored now to fight any number of diseases.

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Protein Vaccine Vaccination Immune Response 40