Pharmaceutical Technology

MARCH 1, 2023

Vertex will also handle all subsequent development, manufacturing, and commercialisation activities. Tevard CEO and co-founder Daniel Fischer said: “Given Vertex’s proven track record of developing novel therapies to treat serious disease, we’re thrilled to partner with them on this important effort. “By

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Development Gene Therapy Genetics Genetic Disease 130

World of DTC Marketing

JANUARY 24, 2022

A drug’s therapeutic target, stage in development, and potential to yield returns shape whether its manufacturer is worth backing — or whether money would be better spent elsewhere. Particularly attractive are medicines ahead of the curve, aimed at diseases likely to move into the spotlight over the next few years. .”

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Life Science Drugs Development Genetic Disease 225

Pharmaceutical Commerce

MAY 5, 2023

New capability will support manufacturing of commercial-stage therapies to help combat cancers and genetic diseases.

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Genetic Disease Production Genetics Manufacturing 52

Pharmaceutical Technology

FEBRUARY 23, 2023

The collaboration will combine the mRNA platform of Moderna with the gene editing technologies suite, including the base editing capabilities of Life Edit for the development of curative therapies to treat challenging genetic diseases. Life Edit will get an upfront payment and is eligible for potential milestone payments.

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Gene Editing Gene In-Vivo Gene Therapy 147

XTalks

MAY 17, 2023

Related: Gene Therapy Clinical Trials for Rare Diseases: 5 Key Takeaways from FDA’s Recent Town Hall “The BGTC offers promise for rare disease patients around the world,” said Julie Gerberding, MD, CEO of the FNIH, in the press release from the agency. In the US, more than 30 million people live with a rare disease.

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Gene Therapy Clinical Trials Clinical Trials Gene 98

pharmaphorum

OCTOBER 26, 2020

Through the acquisition, Bayer will gain full rights to the company’s gene therapy technology and manufacturing platforms, including AskBio’s adeno-associated virus (AAV)-based platform, which has already demonstrated applicability across different therapeutic areas. .

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Gene Therapy Gene Genetic Disease Genetics 59

XTalks

AUGUST 29, 2022

It is difficult to control where the viral vectors insert genes in the genome, and it is difficult to manufacture large quantities of clinical-grade viral vectors. The goal of the team was to significantly increase yields of correctly engineered immune cells with CRISPR to effectively manufacture cell therapies.

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Gene Editing DNA Gene Genome 98

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. As per the agreement, Alexion will initiate a cash tender offer through a subsidiary to acquire all outstanding shares of LogicBio for $2.07

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Gene Editing Gene Genomics Genome 64

pharmaphorum

JANUARY 11, 2021

Under the plans the company’s rare disease drugs will remain under the aegis of bluebird with current genetic disease president Andrew Obenshain taking the reins as CEO. Meanwhile the as-yet unnamed oncology company will spin off under the leadership of bluebird’s current chief executive Nick Leschly.

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Gene Therapy Gene Genetic Disease Manufacturing 66

The Pharma Data

OCTOBER 27, 2020

Agena Bioscience develops, manufactures, and supplies genetic analysis systems and reagents, including the MassARRAY ® System. .” More information is available at www.agenabio.com. About Agena Bioscience. For more information about Agena, visit www.agenabio.com. View original content to download multimedia: [link].

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Genetic Analysis Genome Genomics Reagent 52

Roots Analysis

AUGUST 12, 2024

These diagnostic solutions are essential for detecting and monitoring diseases, identifying genetic abnormalities, and guiding personalized treatment plans. Molecular diagnostic solutions are pivotal across various medical fields, including oncological disorders, infectious diseases, genetic testing, and personalized medicine.

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Genetics Gene Expression RNA DNA 40

XTalks

NOVEMBER 27, 2023

Pompe disease, also referred to as acid-maltase disease and glycogen storage disease II, is an uncommon genetic disorder characterized by the gradual weakening of both cardiac and skeletal muscles. Crowley, executive chairman of Amicus Therapeutics, in the company’s press release. How Do Pombiliti and Opfolda Work?

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Pharmaceutical Technology

JUNE 19, 2023

Pharmaceutical companies are putting their trust in the immense potential this new generation of medicine has for treating individuals with rare genetic diseases, which currently affect an estimated 280 million patients worldwide.

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Gene Therapy Gene Packaging Packaging 130

pharmaphorum

DECEMBER 6, 2020

The trials are the first to test a CRISPR/Cas9 gene editing therapy in humans for a genetic disease, according to the partners. The success of the study is a shot across the bows of companies trying to develop other genetic therapies for thalassaemia and SCD.

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Gene Editing Gene In-Vivo Genetics 52

Roots Analysis

FEBRUARY 21, 2022

The evolution of NGS technologies has made it possible to sequence thousands of genes with a suspected genetic disease predisposition, in a very short period of time and at minimal costs. Nanoparticles Contract Manufacturing. Next Generation Sequencing (NGS) Library Preparation Kits Market – Roots Analysis.

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Marketing Gene Sequencing DNA Genome 52

pharmaphorum

APRIL 20, 2022

Cancer is a genetic disease, caused by certain changes in the way that genes control cell function, such as how they grow and divide. 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?

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Gene Editing DNA Gene Genetics 52

The Pharma Data

DECEMBER 9, 2020

NASDAQ: RCKT) (“Rocket”), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the pricing of an upsized underwritten public offering of 4,642,858 shares of its common stock at a public offering price of $56.00

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Gene Therapy Genetics Genetic Disease Sales 40

The Pharma Data

JANUARY 26, 2021

Strides in anaesthesia and critical care are opening new avenues for surgeons and manufacturers. The advent of biologically active agents has paved way to new treatment avenues in the developmental and genetic disease, thereby boosting the orthopedics market. Orthopedics Market: Regional Assessment.

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Marketing Development Genetic Disease Research 52

XTalks

AUGUST 6, 2020

TSC is a rare genetic disease that affects approximately 1 in 6,000 people. It also provides hope for these patients and their families and is yet another important milestone for Epidiolex as a first-in-class antiepileptic drug.”.

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FDA Approval Protein Containment Genetic Disease 64

XTalks

SEPTEMBER 29, 2021

Yvonne Gwinnell, the senior manager of talent acquisition at Ultra Genyx, which is a biopharmaceutical company involved in the R&D of novel products for the treatment of rare and ultra-rare genetic diseases, shared some of the initiatives her organization took to implement workplace changes with their employees.

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Life Science HR Scientist Genetic Disease 122

Delveinsight

FEBRUARY 11, 2021

Avrobio designed the Plato platform to optimize vector copy numbers, personalize the conditioning regimen, aid automated manufacturing and otherwise promote and industrialize its lentiviral vector-based approach to treating genetic diseases.

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Gene Therapy Gene Immune Response In-Vivo 52

The Pharma Data

DECEMBER 17, 2020

About Lysogene Lysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS). The Company has built a unique capability to enable a safe and effective delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS.

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The Pharma Data

DECEMBER 27, 2020

Lysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS). The Company has built a unique capability to enable a safe and effective delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS. Sarepta Therapeutics, Inc.

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The Pharma Data

OCTOBER 18, 2020

Telomeres degrade and shorten with age and can become excessively damaged in certain genetic diseases, as well as from lifestyle factors such as smoking, poor diet, and chronic stress. Shortening of telomeres is associated with the symptoms of aging, heart disease, DNA damage and uncontrolled cell replication, which can lead to cancer.

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DNA Scientist Genetic Disease Research 52

The Pharma Data

NOVEMBER 2, 2020

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for serious and life-threatening rare and ultra-rare genetic diseases. These risks and uncertainties include, among others: our ability to successfully manufacture vosoritide; the content and timing of decisions by the U.S.

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Drugs Containment Trials Gene 40

The Pharma Data

JANUARY 9, 2021

Hemophilia A, also called Factor VIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. BioMarin is a global biotechnology company that develops and commercializes innovative therapies for serious and life-threatening rare and ultra-rare genetic diseases.

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Gene Therapy Gene Trials Clinical Trials 52

The Pharma Data

OCTOBER 28, 2020

Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. GeneTx licensed the rights to antisense technology intellectual property from the Texas A&M University System in December 2017. About Ultragenyx.

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Protein Production Development Drugs 40

pharmaphorum

OCTOBER 25, 2022

The Japanese firm has agreed to make a $50 million investment in Dallas-based Taysha in exchange for a 15% stake in the company, plus exclusive options to license two clinical-stage, single-gene therapies for rare genetic diseases. ” The post Astellas makes another gene therapy play, takes stake in Taysha appeared first on.

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Gene Therapy Gene Genetic Disease Genetics 104

XTalks

FEBRUARY 24, 2025

Specifically, Ionis said donidalorsen was demonstrated to result in sustained reductions in HAE attack rates while providing enhanced disease control. HAE is a rare genetic disorder characterized by unpredictable, recurrent episodes of severe swelling that can affect various parts of the body, including the hand, feet and airways.

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Trials RNA Allergies Drugs 59

XTalks

MARCH 21, 2024

XTALKS WEBINAR: Cell and Gene Therapy Development: Characterization of Cellular Source Material Live and On-Demand: Tuesday, May 14, 2024, at 11am EDT (4pm BST/UK) Register for this free webinar to learn about characterizing cellular source material for developing and manufacturing cell and gene therapy-based products.

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Gene Therapy Gene Drugs FDA Approval 111

XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

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Gene Therapy Gene Trials Clinical Trials 95

STAT News

FEBRUARY 22, 2023

The Cambridge biotech company announced Wednesday that it will partner with Life Edit Therapeutics to develop potentially permanent treatments for rare genetic diseases and other conditions. Life Edit is the North Carolina subsidiary of ElevateBio, a cell and gene therapy manufacturing firm in Waltham.

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Gene Editing Gene Genetic Disease Research 126

XTalks

DECEMBER 13, 2023

XTALKS WEBINAR: Cryopreservation Best Practices for Cell & Gene Therapy Source Material Live and On-Demand: Monday, January 22, 2024, at 11am EST (4pm GMT/UK) Register for this free webinar to learn about cryopreservation, a vital step in the manufacturing of today’s cell and gene therapies. How do Casgevy and Lyfgenia Work?

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XTalks

DECEMBER 29, 2022

Given their precise targeting, gene therapies have largely been focused on oncology indications followed by rare genetic diseases. As such, most gene therapies in late-stage clinical trials are in oncology or rare disease indications. 3. Manufacturing & Supply Chain: Addressing Drug Shortages.

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Life Science Gene Therapy Gene Manufacturing 52

The Pharma Data

SEPTEMBER 22, 2020

All patients deserve a gene therapy designed to address the genetic root cause of their disease with a single dose. About Spinal Muscular Atrophy SMA is the leading genetic cause of infant death. Zolgensma is approved in the US, Japan and, most recently, Brazil, for patients with SMA under the age of two.

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Clinical Development Gene Therapy Development Gene 52

The Pharma Data

NOVEMBER 24, 2020

PH1 is an ultra-rare genetic disease characterized by oxalate overproduction. About Primary Hyperoxaluria Type 1 (PH1) PH1 is an ultra-rare genetic disease that affects an estimated one to three individuals per million in the United States and Europe. Oxlumo should be administered by a healthcare professional.

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FDA Approval Production RNA Medicine 52

The Pharma Data

DECEMBER 20, 2020

In the rejection, the agency indicated they need to conduct pre-approval inspections at two of scPharmaceuticals’ third-party manufacturing facilities that they couldn’t do because of travel restrictions related to the COVID-19 pandemic. On December 3, 2020, the FDA issued a complete response letter (CRL) for the NDA.

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