Pharmaceutical Commerce

MAY 5, 2023

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

Genetic Disease 52

Genetic Disease Production Genetics Manufacturing 52

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

Pharmaceutical Technology

JANUARY 12, 2023

Under the research partnership deal, the companies will develop new precision genetic medicines for a severe form of genetic dilated cardiomyopathy (DCM). Our pipeline of programmes focused on neuromuscular and cardiac rare disease gives us deep insight into the diverse and complex nature of dilated cardiomyopathy.”.

RNA 130

RNA Gene Therapy Genetics Genetic Disease 130

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.

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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. .

Gene Therapy 59

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.

Gene Editing 98

Gene Editing DNA Gene Genome 98

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.

Gene Therapy 66

Gene Therapy Gene Genetic Disease Manufacturing 66

pharmaphorum

OCTOBER 4, 2022

Through Alexion , which AstraZeneca acquired in 2021, the company will gain access to LogicBio’s technology platforms for the delivery and insertion of genes to address genetic diseases, as well as a platform designed to improve viral vector manufacturing processes. per share, a rare 660% premium on LogicBio’s share price.

Gene Editing 64

Gene Editing Gene Genome Genomics 64

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.

Gene Therapy 130

Gene Therapy Gene Packaging Packaging 130

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.

Marketing 52

Marketing Gene Sequencing DNA Genomics 52

The Pharma Data

OCTOBER 27, 2020

Agena Bioscience develops, manufactures, and supplies genetic analysis systems and reagents, including the MassARRAY ® System. Agena’s MassARRAY SARS-CoV-2 Panel kits and instruments are ready for immediate deployment, and we are equipped to supply millions of tests each month.” About Agena Bioscience.

Genetic Analysis 52

Genetic Analysis Genome Genomics Reagent 52

XTalks

NOVEMBER 27, 2023

This recombinant human α-glucosidase (rhGAA) is manufactured using a perfusion methodology involving a Chinese hamster ovary (CHO) cell line, resulting in N-glycans of CHO cell origin. How Do Pombiliti and Opfolda Work? Pombiliti is a specialized hydrolytic enzyme with a specific affinity for lysosomal glycogen.

FDA Approval 104

FDA Approval Clinical Trials Clinical Trials Trials 104

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.

Marketing 52

Marketing Development Genetic Disease Research 52

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. Similarly, three SCD patients suffered none of the characteristic painful attacks – known as vaso-occlusive crises (VOCs) – in the three to 15-month follow-up period after the treatment.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 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.”.

FDA Approval 64

FDA Approval Protein Containment Genetic Disease 64

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. This approach would greatly simplify the manufacturing process and also allow the treatments to be provided quickly to patients in need. Why cancer?

Gene Editing 52

Gene Editing DNA Gene Genetics 52

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

The Pharma Data

DECEMBER 9, 2020

Rocket intends to use the net proceeds from this offering to further fund the development of its pipeline of gene therapies for rare diseases, including filing for marketing authorization for RP-L201 in the United States and Europe, accelerating the buildout of in-house manufacturing capabilities, and for general corporate purposes.

Gene Therapy 40

Gene Therapy Genetics Genetic Disease Sales 40

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.

Gene Therapy 52

Gene Therapy Gene Immune Response In-Vivo 52

Roots Analysis

AUGUST 12, 2024

Genetic Diseases – One of the primary applications of molecular diagnostic solutions is to identify genetic mutations and variations associated with inherited disorders, enabling early detection, carrier screening, and personalized treatment approaches for genetic diseases.

Genetics 40

Genetics Gene Expression RNA DNA 40

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.

DNA 52

DNA Scientist Genetic Disease Research 52

The Pharma Data

DECEMBER 17, 2020

Lysogene is also collaborating with an academic partner to define the strategy of development for the treatment of Fragile X syndrome, a genetic disease related to autism. In accordance with the agreements signed between Lysogene and Sarepta Therapeutics, Inc., Sarepta Therapeutics, Inc. www.lysogene.com.

Gene Therapy 40

Gene Therapy Clinical Trials Clinical Trials Gene 40

The Pharma Data

DECEMBER 27, 2020

Lysogene is also collaborating with an academic partner to define the strategy of development for the treatment of Fragile X syndrome, a genetic disease related to autism. In accordance with the agreements signed between Lysogene and Sarepta Therapeutics, Inc., Sarepta Therapeutics, Inc. www.lysogene.com. Forward Looking Statement.

Gene Therapy 40

Gene Therapy Clinical Trials Clinical Trials In-Vivo 40

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.

Drugs 40

Drugs Containment Trials Marketing 40

The Pharma Data

JANUARY 9, 2021

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for serious and life-threatening rare and ultra-rare genetic diseases. Approximately 1 in 10,000 people have Hemophilia A. About BioMarin. For additional information, please visit www.biomarin.com.

Gene Therapy 52

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.

Protein 40

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

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

FEBRUARY 24, 2025

XTALKS WEBINAR: Nanopore Sequencing for RNA Vaccines and Therapeutics: Advancing Drug Development and Quality Control Live and On-Demand: Wednesday, March 26, 2025 , at 11am EDT (4pm CET / EU-Central) Register for this free webinar to gain insights into how nanopore sequencing is poised to become a key player in the biopharma industry and how it can (..)

Trials 59

Trials RNA Allergies Drugs 59

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.

Gene Editing 126

Gene Editing Gene Genetic Disease Research 126

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.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

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.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

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?

Gene Therapy 98

Gene Therapy Gene FDA Approval In-Vivo 98

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.

In-Vitro 52

In-Vitro FDA Approval Drugs Manufacturing 52

The Pharma Data

NOVEMBER 8, 2020

The New Drug Application (NDA) for the drug is based on data from four Phase III trials in pediatric patient populations from the age of 6 to 17 years, two Phase II trials, several Phase I trials, a long-term open label extension study, preclinical testing, and drug manufacturing data. Adamis Pharmaceuticals’ Zimhi for Opioid Overdose.

Trials 52

Trials Protein Gene Genetic Disease 52

The Pharma Data

AUGUST 3, 2021

SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement. Our robust AAV-based pipeline is advancing treatments for Rett syndrome and Friedreich’s ataxia.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Gene 52