Scienmag

JUNE 10, 2025

Cell lines often lose critical tumor-specific features through prolonged in vitro culture, but PDXs maintain the malignant phenotype in vivo, providing a more faithful and clinically relevant experimental framework. Despite their promise, PDX models confront substantial hurdles that have limited their widespread adoption.

Research 59

Research Clinical Trials Clinical Trials Gene Editing 59

Pharmaceutical Technology

FEBRUARY 23, 2023

DNA binding site prediction, peptide structure optimisation, and AI-assisted genome analysis are some of the accelerating innovation areas, where adoption has been steadily increasing. However, not all innovations are equal nor do they follow a constant upward trend.

Drugs 189

Drugs DNA In-Vivo In-Vitro 189

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.

In-Vivo 52

In-Vivo Gene Editing Trials Gene 52

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.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

XTalks

AUGUST 5, 2020

Rosha Poudyal, PhD, Science and Technology Advisor at 10x Genomics, discussed some of the innovative single cell technology tools that the company is developing and their application in various research areas including oncology, infectious disease and immunology. The Power of Single Cell Technology. Feature barcoding using gel beads.

Gene Expression 112

Gene Expression Development RNA Drugs 112

XTalks

MAY 22, 2024

John Finn, PhD Chief Scientific Officer Tome Biosciences Dr. Finn has over 20 years of experience in the gene therapy space with a focus on genome editing and delivery technologies. PGI is a cutting-edge gene editing technology that allows for the insertion of large sequences of DNA with site-specific precision.

Gene Editing 52

Gene Editing Life Science Gene Gene Therapy 52

Scienmag

JUNE 5, 2025

The story began when a young boy from Alabama underwent comprehensive whole-genome sequencing, which revealed a mutation in the VMA21 gene. These observable defects underscore the profound effect that VMA21 mutations exert on muscle structure and function in vivo.

Genetics 59

Genetics Gene Genomics Genome 59

The Pharma Data

JANUARY 12, 2021

KSQ’s proprietary CRISPRomics® discovery engine enables genome-scale, in vivo validated, unbiased drug discovery across broad therapeutic areas. KSQ was founded by thought leaders in the field of functional genomics and pioneers of CRISPR screening technologies. and retain royalties on all ex-U.S. sales for that product.

Development 52

Development In-Vivo Research Genomics 52

The Pharma Data

DECEMBER 10, 2020

ImmunityBio has engineered the vector to overcome this problem and has shown that its second-generation adenovirus vector can safely and effectively deliver its cargo even in patients with pre-existing adenovirus immunity. This blocking of viral replication was observed in both the lung and nasal passages.

Vaccine 52

Vaccine Vaccination Immune Response Protein 52

The Pharma Data

JANUARY 4, 2021

Kiromic chPD1 has shown in preclinical data to show a cytotoxic response in 9 different in vivo models with 100% long-term PFS with the induction of host memory responses. chPD1 will be used in the Company’s proprietary chimeric antigen receptor therapy (CAR-T) platform using gamma-delta T-cells (GD-T). About Kiromic.

In-Vivo 40

In-Vivo Development Immune Response Big Data 40

pharmaphorum

JANUARY 29, 2021

Genome editing is an exciting but still nascent field, and companies in the area face as many obstacles as they do opportunities. Sangamo CEO Sandy Macrae told us how his company is being cautious about the hype and finding ways to be financially viable in an emerging space. Zinc fingers. billion in funding.

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

Pharmaceutical Technology

FEBRUARY 15, 2023

Innovation S-curve for the pharmaceutical industry Gene splicing using nucleases is a key innovation area in the pharmaceutical industry Nucleases play a fundamental role in the field of recombinant DNA technology, or genetic engineering. However, not all innovations are equal and nor do they follow a constant upward trend.

Gene Splicing 130

Gene Splicing Gene Recombinant DNA Technology DNA 130

Pharmaceutical Technology

MARCH 9, 2023

Innovation S-curve for the pharmaceutical industry Transgenic murine models is a key innovation area in pharmaceutical s Transgenic murine models refer to mice that have been genetically altered for the purposes of understanding the in vivo functions of genes.

In-Vivo 130

In-Vivo Antibody Genetics Gene 130

pharmaphorum

OCTOBER 8, 2020

Doudna is serving as scientific advisor to Scribe, and its chief executive is Benjamin Oakes, who has been working on genome editing for most of his scientific career, starting out looking at a rival technology to CRISPR called zinc finger nucleases before working in Doudna’s lab at the University of California, Berkeley. Benjamin Oakes.

Gene Editing 69

Gene Editing Gene In-Vivo Clinical Trials 69

Delveinsight

FEBRUARY 25, 2021

Instead of snipping the genome, base editing lets for edits of individual letters in a genetic sequence. Electroporation is used for ex vivo delivery of therapies to blood and immune cells. Beam announced that it paid USD 120 million upfront to acquire Guide Therapeutics in an all-stock agreement.

DNA 52

DNA In-Vivo Genetics Medicine 52