Drug Discovery World

APRIL 17, 2024

DDW’s Diana Spencer speaks to Josh Mandel-Brehm , CEO of Massachusetts-based biotech CAMP4, to understand the role regulatory RNAs play in gene expression and how the founders overcame the challenge of launching a business based on brand new science. What are the advantages compared to other gene modulating approaches?

Gene Expression 59

Gene Expression Gene Genetic Disease RNA 59

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.

Drugs 52

Drugs Gene Editing Genome Genomics 52

Delveinsight

JULY 24, 2021

In addition to this, Angelman syndrome pipeline also involves gene therapy candidates such as GTX-101 which is a therapy of GeneTx Biotherapeutics. Another therapy by the same company named GTX-102 is also an antisense therapy; however, it silences gene expression by binding to the RNA molecule that regulates its expression.

Gene Therapy 96

Gene Therapy Gene Expression Gene RNA 96

XTalks

MARCH 13, 2024

Biomarker testing is being pursued to identify biological signs of the disease and provide an approach to develop personalized treatment plans for each patient. As this technology continues to evolve, it is expected to help identify glaucoma in its nascent stages of development.

Gene Therapy 111

Gene Therapy Gene Life Science DNA 111

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.

Gene Therapy 52

Gene Therapy Gene Scientist Gene Editing 52

XTalks

NOVEMBER 6, 2020

Canadian clinical-stage biotech company Symvivo Corporation has developed an oral COVID-19 vaccine that entered clinical trials this week. The gene therapy-based vaccine targets mucosal tissues in the gastrointestinal tract, allowing for greater selectivity and targeted delivery compared to traditional vaccines.

Clinical Trials 75

Clinical Trials Clinical Trials Vaccination Vaccine 75

pharmaphorum

JANUARY 10, 2023

Astellas has licensed rights to a drug developed by Selecta Biosciences that could make more patients eligible for treatment with its gene therapy for inherited neuromuscular disorder Pompe disease. The post Astellas licenses companion drug for Pompe gene therapy appeared first on.

Gene Therapy 112

Gene Therapy Gene Licensing Licensing 112

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. Genetic literacy among clinicians may not be as high as you think.

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

Roots Analysis

JANUARY 14, 2024

With the increased interest and gradual shift of investment from small molecule drugs to biologics and the establishment of several biologics manufacturing companies / biologics CMOs, more than 250 biologic therapies and vaccines have been developed, globally.

Contract Manufacturing 40

Contract Manufacturing Manufacturing Protein Bacteria 40

Drug Discovery World

FEBRUARY 10, 2023

Scandinavia represents one of the most exciting but perhaps underrepresented markets for the discovery and development of pharmaceuticals. Sweden’s place in the market Scandinavia is home to some of the strongest research and development hubs, with highly respected academic institutes that are helping drive the sector forward.

Life Science 52

Life Science Drugs Gene Therapy Manufacturing 52

The Pharma Data

JULY 12, 2021

.” In the new study, Ghosh and colleagues replaced the first and last steps in preclinical drug discovery with two novel approaches developed within the UC San Diego Institute for Network Medicine (iNetMed), which unites several research disciplines to develop new solutions to advance life sciences and technology and enhance human health.

Drugs 52

Drugs Gene Expression Big Data Gene 52

Roots Analysis

AUGUST 31, 2023

Overview of Gene Switch The notion that genes might be turned on and off was discovered several decades ago when studies revealed that E. Gene switches are sites on genes where regulatory molecules can bind to trigger transcription process, leading to expression of a particular gene.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

The Pharma Data

OCTOBER 15, 2020

Immuno-oncology and CAR T cells energized the field of regenerative medicine, but for cell and gene to deliver on their promises, new, disruptive technologies and new modes of operation are needed. Other cell and gene companies are advancing solutions, too. It’s not a quick fix,” he said. Source link.

Manufacturing 52

Manufacturing Gene Therapy Gene Gene Expression 52

Pharmaceutical Technology

JANUARY 24, 2023

The investigational adeno-associated virus (AAV) gene therapy candidate NGN-401 is claimed to be the first to deliver the full-length human MECP2 gene using the company’s Expression Attenuation via Construct Tuning (EXACT) gene regulation technology.

Gene Therapy 208

Gene Therapy Gene Gene Expression Regulation 208

The Pharma Data

NOVEMBER 8, 2020

BOSTON–( BUSINESS WIRE )– Decibel Therapeutics , a clinical-stage biotechnology company developing novel restorative gene therapeutics to treat hearing loss and balance disorders, today announced the closing of an oversubscribed Series D financing, which raised $82.2M. 9, 2020 11:00 UTC. partner at OrbiMed.

Gene Therapy 40

Gene Therapy Gene Gene Expression Genome 40

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.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

Drug Discovery World

FEBRUARY 29, 2024

They are called: Sitting down with… PharmEnable , Is single-cell gene expression the next trend in next generation sequencing? In the third article, Samsung Biologics’ Senior Vice President, James Park, explores the opportunities emerging for CDMOs to support mRNA developers as they explore new therapeutic areas for patients.

Gene Expression 52

Gene Expression DNA Genome Genomics 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. It’s a great tool for controlling gene expression.”.

DNA 98

DNA Gene Gene Silencing Genome 98

Delveinsight

JANUARY 18, 2021

The first sign of a child suffering from Angelman syndrome is a delay in development, such as the inability to sit without support or making incoherent babbling sounds. Angelman syndrome is a result of the missing or dysfunctional gene known as UBE3A. GeneTx and Ultragenyx are co-developing the therapy.

Marketing 52

Marketing Gene Drugs Protein 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 without editing the genetic sequence itself. These modifications regulate gene expression without altering the sequence or structure of DNA.

DNA 52

DNA Gene Gene Silencing Genome 52

Drug Discovery World

APRIL 30, 2024

Reece Armstrong explores the potential and future opportunities for mRNA-based therapies. Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. However, these therapies can cause severe adverse effects.

RNA 52

RNA Protein Vaccination Vaccine 52

Drug Discovery World

OCTOBER 17, 2023

BN : Our bodies consist of an incredibly complex network of about 35 trillion cells divided into as many as 300 cell types that fulfil distinct roles in development, homeostasis, and disease. This limits its efficacy in later stages of drug development or in the discovery and characterisation of biomarkers.

Gene Expression 52

Gene Expression Drugs Protein Gene 52

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. Adult fibroblasts were cultured in a cocktail of transcription factors to reactivate embryonic gene expression patterns 2.

Gene Editing 52

Gene Editing Gene Engineer DNA 52

Drug Discovery World

JULY 18, 2023

Since its discovery in 2012, CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) technology has revolutionised the biomedicine and cell and gene therapy fields, providing a versatile tool for precise and efficient genome editing. CRISPR diagnostics have successfully detected various pathogens, including RNA viruses (eg.

DNA 98

DNA Genome Genomics RNA 98

The Pharma Data

DECEMBER 27, 2020

The drug is a humanized IgG4 monoclonal antibody that blocks CCR5 and is being developed for COVID-19, HIV and metastatic triple-negative breast cancer. TSHA-101 is an investigational gene therapy designed to deliver the HEXA and HEXB genes driven by a single promoter within the same AAV9 construct, dubbed a bicistronic vector.

Trials 52

Trials Antibody Gene Therapy Clinical Trials 52

XTalks

DECEMBER 24, 2020

Focusing on biologics, vaccine development and policy platforms in anticipation of an eventual outbreak is why we’re where we are today in terms of the positive side [of the pandemic].”. Most antibody drugs and vaccines have been developed to target parts of the spike protein. Pandemic Efforts.

Life Science 111

Life Science Vaccination Vaccine Gene 111