XTalks

NOVEMBER 3, 2023

After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene therapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. CRISPR works as genetic scissors to edit parts of the genome.

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Gene Therapy Gene Gene Editing In-Vivo 59

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., a biotechnology company specializing in the development of lipid nanoparticle (LNP) delivery systems for molecular therapeutics.

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Gene Therapy Gene Gene Editing In-Vivo 40

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.

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Drug Discovery World

FEBRUARY 27, 2023

An investigational gene therapy for Sanfilippo syndrome – which leads to a form of childhood dementia – has shown promising early results in a proof-of-concept study. The post Gene therapy for ‘childhood dementia’ shows promise appeared first on Drug Discovery World (DDW).

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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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Gene Editing In-Vivo Gene Sequencing Gene 88

Drug Discovery World

OCTOBER 6, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

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Drugs Gene Editing Genome Genomics 52

The Pharma Data

JANUARY 12, 2021

Marianne De Backer, Head of Business Development & Licensing in Bayer’s Pharmaceuticals Division, pictured above. Also in December, the company partnered with Atara Biotherapeutics to develop off-the-shelf T-cell immunotherapies for high mesothelin-expressing tumors. Photo courtesy of Bayer. We have set the bar high.

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Drug Discovery World

DECEMBER 21, 2023

DDW’s top 10 most popular articles for 2023 reflect the key subjects in drug discovery and development. In a record year for gene therapy approvals, it isn’t surprising that cell & gene therapy (CGT) has been a hot topic. Read the article – Expert view: What’s next for cell and gene therapy?

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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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Drug Discovery World

JUNE 22, 2023

“This is part of our long-term business strategy of leveraging exosome-mediated delivery to create and enable genetic medicines such as gene therapy and genome editors”.

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Gene Therapy Gene Editing Drug Delivery Gene 52

Drug Discovery World

OCTOBER 10, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

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Gene Editing Genome Genomics Genetics 59

Drug Discovery World

MAY 6, 2024

In this article, DDW’s Diana Spencer highlights several prominent hotspots that are taking the lead on advancing European drug discovery and development. Even before this, however, Amsterdam was established as an important centre for drug development. The CDD will sponsor and manage KWF-supported trials.

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Drugs Life Science Gene Editing Gene 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., as Head of Business Development. .–( BUSINESS WIRE )– EdiGene, Inc. He received his B.S. He received his B.S.

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Drug Discovery World

JULY 10, 2023

If there’s one thing I would take from the Summer 2023 issue of Drug Discovery World , it would be that the application of technologies and processes throughout the pharmaceutical industry are changing how scientists pursue the development of therapies. There are myriad ways that this is being enabled.

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XTalks

NOVEMBER 30, 2023

Global entities such as the WHO, the Global Fund and the Joint United Nations Programme on HIV/AIDS (UNAIDS) have developed comprehensive strategies aligned with Sustainable Development Goal (SDG) target 3.3, These findings signify a significant step forward in the development of AGT103-T as a potential gene therapy for HIV.

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Drug Discovery World

OCTOBER 16, 2023

Alchemab, on the other hand, has developed a highly differentiated platform which enables the identification of novel drug targets and therapeutics by analysis of patient antibody repertoires, drawing from the expertise in Cambridge to progress the company’s mission to build a broad pipeline of protective therapeutics for hard-to-treat diseases.

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XTalks

DECEMBER 14, 2022

Expanding upon the CRISPR-Cas9 gene editing system, researchers at MIT have designed a new technique called PASTE gene editing that can cut out defective genes and replace them with new genes in a safer and more efficient way. The PASTE gene editing technique was recently published in Nature Biotechnology.

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Gene Editing DNA Gene Gene Sequencing 98

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.

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Genetics Medicine Gene Therapy Marketing 52

Cloudbyz

MARCH 6, 2024

The Evolution of Pharmaceutical Therapies: The emergence of biologics represents a significant paradigm shift in the pharmaceutical industry, prompting a reevaluation of traditional drug development and manufacturing processes. Challenges and Future Directions: Despite their immense promise, biologics are not without challenges.

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Gene Immune Response Manufacturing Gene Therapy 72

pharmaphorum

AUGUST 31, 2022

After a recent approval, there are now three gene therapies available on the US market. In recent years, gene therapy has transitioned from a promising idea to a reality for patients, with many of the severe safety issues that emerged in early iterations of the technology being overcome. from 2021 to 2029.

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Gene Therapy Gene Gene Editing DNA 91

Drug Discovery World

DECEMBER 15, 2022

As gene editing technologies like CRISPR progress toward clinical study, researchers must continue to advance new approaches and address inherent challenges, explains Jon Chesnut, PhD, Senior Director, Cell Biology R&D, Thermo Fisher Scientific. Early phase clinical trials for gene editing therapies.

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Gene Editing Gene In-Vivo Genome 52

Delveinsight

SEPTEMBER 14, 2021

ADARx Pharmaceuticals, a biotechnology company developing RNA targeting therapeutics , announced the completion of a USD 75 million Series B financing to progress its drug development pipeline. VOR33 comprises CRISPR genome-edited hematopoietic stem and progenitor cells , which have been engineered to exclude CD33.

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RNA Clinical Trials Clinical Trials Drugs 67

The Pharma Data

NOVEMBER 3, 2020

According to the FDA, the purpose of Fast Track designation is to get important new drugs to patients earlier by facilitating the development, and expediting the review, of drugs to treat serious conditions and fill an unmet medical need. 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 52

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. Meanwhile, others are researching the link between gene variation and different responses to treatments.

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Drug Discovery World

JANUARY 26, 2023

DDW Editor Reece Armstrong takes a look at the pharmaceutical research and development market in 2023 and asks key opinion leaders what challenges and trends will emerge throughout the year. billion showed the pharma giant taking further steps into the oncology market and developing its speciality medicines vaccines portfolio.

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Drug Discovery World

MARCH 9, 2023

The post FDA clears in vivo gene editing therapy for trials in US appeared first on Drug Discovery World (DDW). The US Food and Drug Administration (FDA) has cleared Intellia Therapeutics’ Investigational New Drug (IND) application for NTLA-2002 for the treatment of hereditary angioedema (HAE).

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XTalks

DECEMBER 20, 2023

As we step into 2024, the life sciences continue to evolve at an unprecedented pace, driven by technological innovation, a deeper understanding of human biology and the application of new technologies in areas like drug development and health wearables. Regulatory bodies are also taking note of the applications of AI in drug development.

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Life Science Gene Editing Development Clinical Trials 119

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. In 2020, 31 out of 53 novel drug approvals were for rare or orphan diseases​​.

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Research Drugs Gene Therapy Clinical Trials 59

Drug Discovery World

JULY 18, 2023

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. Years of development and fast-paced research have continued to unlock its potential, expanding how CRISPR can be used to treat, detect, and prevent common diseases such as cancer and Covid-19.

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DNA Genome Genomics RNA 98

Drug Discovery World

JANUARY 23, 2023

Kevin Hemphill, R&D Manager at PerkinElmer’s Horizon Discovery explores how base editing has emerged as an attractive gene editing option for researchers wanting to develop stem cell-based therapies. In addition, iPSC lines, despite their pluripotency, have skewed suitability towards certain cell types 12,13.

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Gene Editing Gene Engineer DNA 52

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. Epigenetic Editing with CRISPR.

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DNA Gene Gene Silencing Genome 98

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. Epigenome Editing with CRISPR.

DNA 52

DNA Gene Gene Silencing Genome 52

Drug Discovery World

MAY 30, 2023

From antibody-drug conjugates and RNA, to CRISPR and CAR-T therapy, DDW asked the drug discovery industry what the most important development in oncology over the last five years has been. Immunopheresis is a ‘subtractive therapy,’ in contrast to alternative drugs that constitute ‘additive’ therapies.

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Research Drugs Protein RNA 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.

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XTalks

DECEMBER 24, 2020

From isolating SARS-CoV-2 in early January to sequencing its genome shortly thereafter and having a prototype vaccine against it within days, scientific process and progress have held steadfast throughout the pandemic. Infectious disease expert, epidemiologist and director of the Yale Institute for Global Health at Yale University, Saad B.

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Pharmaceutical Technology

FEBRUARY 20, 2023

This may sound obvious, but it is still a major hurdle in drug development. This may lead to therapies and dosing regimes being less suitable for minority populations – and according to Hans Bunschoten, chief strategy officer at clinical research company Cerba Research, part of Cerba Healthcare, this is a major problem.

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