pharmaphorum

SEPTEMBER 12, 2022

Alongside drugs and therapeutics, data collection and technology enhancements have redefined the traditional healthcare experience, especially within oncology, and genomic profiling has become a significant factor in allowing for personalised care. Tailored oncology. In other words, every person’s cancer has its own genetic construct.

Genome 105

Genome Genomics Clinical Trials Clinical Trials 105

Pharmaceutical Technology

MARCH 8, 2023

Its focus areas include human systems, precision diagnostics, data sciences, next-generation biomanufacturing, and genomic medicines. This Beacon’s focus will be on product solutions that help address the bottlenecks impacting manufacturing yield and quality of cell therapies.

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Gene Therapy Manufacturing Gene Production 244

Scienmag

JANUARY 19, 2021

publishers New Rochelle, NY, January 19, 2021–Gene editing therapies, including CRISPR-Cas systems, offer the potential to correct mutations causing inherited retinal degenerations, a leading cause of blindness. Credit: Mary Ann Liebert, Inc.,

Gene Editing 101

Gene Editing Genome Genomics Gene Therapy 101

Cloudbyz

JULY 31, 2024

The landscape of modern medicine is rapidly evolving, driven by groundbreaking advancements in diagnostics. This revolution is enabling the growth of innovative biomarker-based precision medicine and cell and gene therapy, transforming both clinical research and post-market care.

Gene Therapy 52

Gene Therapy Gene Medicine In-Vitro 52

Scienmag

JULY 23, 2021

Jude Children’s Research Hospital study highlights the power of comprehensive whole genome, whole exome and RNA sequencing to better understand and treat each patient’s cancer Credit: St. Jude Children’s Research Hospital St.

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Medicine Genome Genomics RNA 96

BioSpace

JUNE 24, 2024

Exsilio Therapeutics emerged from stealth on Tuesday with a platform that leverages mRNA technology to develop redosable genomic medicines for a range of complex diseases.

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Gene Therapy Genome Genomics Gene 66

pharmaphorum

NOVEMBER 12, 2020

UCB has made a new foray into the gene therapy space, buying fellow Belgium-based company Handl to get control of its adeno-associated virus (AAV) capsid delivery platform and two research programmes in neurodegenerative diseases. The post UCB revs up its gene therapy drive with Handl acquisition appeared first on.

Gene Therapy 52

Gene Therapy Gene Manufacturing Contract Manufacturing 52

Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna Genomics general manager and chief scientific officer Eric Huang said: “Through our collaboration with Life Edit, we hope to harness the power of gene editing technologies as part of our broader research and development engine, helping to advance our mission and deliver on the promise of mRNA.”

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Gene Editing Gene In-Vivo Gene Therapy 147

Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

Gene Therapy 52

Gene Therapy Gene Production Clinical Trials 52

BioSpace

OCTOBER 20, 2020

Paris-based SparingVision, a genomic medicine company focused on ocular diseases, raised €44.5 million (approximately $52.2 million) in a financing round.

Gene Therapy 52

Gene Therapy Genome Genomics Gene 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.

Gene Editing 64

Gene Editing Gene Genome Genomics 64

FDA Law Blog

NOVEMBER 2, 2023

Lewis, Senior Regulatory Device & Biologics Expert — On October 20, 2023, FDA announced the availability of the final guidance authored by CBER titled “Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies.” It finalized a draft guidance published in 2022.

Medicine 59

Medicine Gene Therapy Gene Production 59

Pharmaceutical Technology

JUNE 29, 2022

The US Food and Drug Administration (FDA) put a high-profile bluebird bio trial for sickle cell disease on partial clinical hold, and advisory panels deliberated over decisions involving gene therapies for amyotrophic lateral sclerosis (ALS), cerebral adrenoleukodystrophy (CALD), and beta-thalassemia.

Drugs 246

Drugs FDA Approval Development Marketing 246

pharmaphorum

DECEMBER 8, 2022

The advent of biologic therapies in the 1970s along with the decoding of the human genome in 2003 heralded an explosion in pharmaceutical innovation. Additionally, new forms of data, such as real‑world evidence (RWE), are becoming more readily accepted for demonstrating the long‑term value of novel medicines.

Big Data 96

Big Data Gene Therapy Medicine Marketing 96

Scienmag

JANUARY 27, 2021

— Researchers used single-molecule imaging to compare the genome-editing tools CRISPR-Cas9 and TALEN. Their experiments revealed that TALEN is up to five times more efficient than CRISPR-Cas9 in parts of the genome, called heterochromatin, that are densely packed. Credit: Composite photo by L. Brian Stauffer CHAMPAIGN, Ill.

Genome 52

Genome Genomics DNA Research 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

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Scienmag

NOVEMBER 12, 2020

In a study that has unprecedented implications to advance both medicine and biodiversity conservation, researchers have sequenced 131 new placental mammal genomes, bringing the worldwide total to more than 250 In a study that has unprecedented implications to advance both medicine and biodiversity conservation, researchers have sequenced 131 new placental (..)

Biobanking 41

Biobanking Medicine Genome Genomics 41

Scienmag

FEBRUARY 21, 2021

‘Exploring one of the biggest challenges facing genomics today – understanding genetic variants’ SEATTLE (February 22, 2021) – Global experts performing leading edge research in precision medicine, functional genomics, protein science, and variant interpretation and prediction will share their expertise and insights April 5-7 (..)

Genome 40

Genome Genomics Genetics Protein 40

The Pharma Data

NOVEMBER 8, 2020

DB-OTO, Decibel’s investigational gene therapy to restore hearing in children with congenital deafness due to a deficiency in the otoferlin gene, is in preclinical studies, and Decibel expects to initiate clinical testing in 2022. Thompson, M.D., a partner at OrbiMed, to its board of directors. partner at OrbiMed.

Gene Therapy 40

Gene Therapy Gene Gene Expression Genome 40

Pharma Marketing Network

DECEMBER 21, 2020

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

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

Scienmag

MARCH 11, 2021

The CRISPR-based gene editor, C-to-G Base Editor (CGBE), opens up treatment avenues for up to 40 per cent of genetic disorders caused by single-nucleotide mutations Credit: Agency for Science, Technology and Research (A*STAR), Genome Institute of Singapore (GIS) A team of researchers from the Agency for Science, Technology and Research’s (A*STAR) (..)

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Gene Scientist Genome Genomics 74

XTalks

JANUARY 2, 2024

For instance, Vyjuvek , the first FDA-approved gene therapy for DEB, is priced at $24,250 per vial. a biotech company specializing in the development and commercialization of genetic medicines for rare diseases, announced FDA approval for Vyjuvek for the treatment of DEB.

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FDA Approval Gene Therapy Gene Trials 111

Cloudbyz

JUNE 26, 2023

This can aid in personalized medicine, disease management, and risk assessment, leading to improved patient care and outcomes. Generative AI has the potential to transform clinical research by leveraging large-scale data analysis, simulating complex biological systems, and facilitating personalized medicine.

Clinical Research 97

Clinical Research Research Clinical Trials Clinical Trials 97

Pharmaceutical Technology

FEBRUARY 28, 2023

The next generation of orphan drug launches Looking at some of the more recent orphan drug approvals, it is clear that advancements in genomic medicine are now shaping the next generation. By doing so, we not only help patients, but also support manufacturers to further invest in rare disease medicines.”

Medicine 130

Medicine Marketing Production Drugs 130

pharmaphorum

JANUARY 24, 2023

Though instances of this kind of therapy are rare, it is a growing area. The cost of testing per human genome in 2006 was approximately $14 million , and in less than two decades, an average consumer-purchased genetic test costs $100. The information gathered from the project is still providing insights today, a decade later.

Genetics 98

Genetics Drugs Genome Project Biobanking 98

pharmaphorum

JULY 4, 2022

And this is where modern technologies and data-driven medical informatics can really bridge the gaps in rare disease research,” said Dr Joanne Hackett, head of Genomic and Precision Medicine at IQVIA, during a recent pharmaphorum webinar. Thus, she belongs to the Center for Medical Informatics at the University Medicine Dresden.

Genome 105

Genome Genomics Research Medicine 105

XTalks

DECEMBER 20, 2023

Some notable ones include: Editas Medicine : Focused on developing therapies for genetically defined diseases, Editas Medicine is working on CRISPR-based treatments for conditions such as Leber congenital amaurosis (a form of inherited blindness) and blood disorders.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

Camargo

DECEMBER 10, 2021

In October 2017, the European Commission (EC) and the European Medicines Agency (EMA) collaborated with the Member States’ authorities to launch a plan to reduce discrepancies across the EU in applying certain legislation to advanced therapy medicinal products (ATMPs) containing or consisting of genetically modified organisms (GMOs).

Development 176

Development Genetics Production Clinical Trials 176

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.

Research 59

Research Drugs Gene Therapy Clinical Trials 59

pharmaphorum

MAY 18, 2022

Genomics has unlocked entirely new insights to what exactly may cause certain neurological conditions. Also, there is particular emphasis on exploring the potential of gene therapy to develop targeted treatments for these and further neurodegenerative conditions. billion in 2021.

Gene Therapy 92

Gene Therapy Gene Genetics Development 92

Roots Analysis

FEBRUARY 21, 2022

Since the introduction of a gene sequencing method by Frederick Sanger in 1977, the field of genomic data collection and analysis has evolved significantly. Advancement in DNA sequencing technologies have resulted in noteworthy developments in various healthcare-related research fields, such as diagnostics and personalized medicine.

Marketing 52

Marketing Gene Sequencing DNA Genome 52

Scienmag

MAY 26, 2021

Skokie, IL – The International Society for Stem Cell Research (ISSCR), today released updated guidelines for stem cell research and its translation to medicine. The update reflects emerging advances including, stem cell-based embryo models, human embryo research, chimeras, organoids, and genome editing.

Research 45

Research Genome Genomics Medicine 45

pharmaphorum

OCTOBER 13, 2022

At the end of last year, the company partnered with Atara on mesothelin-targeted CAR-T cell therapies for solid tumours. Bayer bought Vividion for $1.5 The post Bayer’s Vividion unit forges $930m cancer alliance with Tavros appeared first on.

Gene Therapy 64

Gene Therapy Genome Genomics Drugs 64

Pharmaceutical Technology

FEBRUARY 3, 2023

The field of genomic medicine has reached a true turning point. With scientists fervently developing mRNA vaccines, nucleic acid therapeutics, and viral vector-based gene therapies, clinicians are set to have a growing number of tools available to treat a wide range of conditions, from infectious diseases to genetic disorders and more.

Genome 244

Genome Genomics Medicine Gene Therapy 244

pharmaphorum

AUGUST 3, 2021

Recent advances in medical science, from genomics and gene therapies to artificial intelligence (AI), have transformed the healthcare landscape, and triggered a review of NICE’s methods and processes. There are now so many more medicines for rare diseases and other smaller patient populations. “We

Medicine 59

Medicine Genome Genomics Genetics 59

The Pharma Data

DECEMBER 13, 2020

which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., Prior to joining EdiGene, he was Vice President of KLUS Pharma and focused on cell therapy and new technologies. 14, 2020 10:00 UTC.

Business Development 40

Business Development Development Gene Editing In-Vivo 40

Delveinsight

SEPTEMBER 14, 2021

CEO of SR One, said that the ADARx team focuses on expeditiously translating their insights around RNA biology into novel medicines to benefit patients. Vor Biopharma has declared that the FDA has awarded their acute myeloid leukemia (AML) engineered hematopoietic stem cell therapy (eHSC), VOR33, a Fast Track Designation.

RNA 67

RNA Clinical Trials Clinical Trials Drugs 67