Pharmaceutical Technology

APRIL 28, 2023

EDIT-301 is made of patient-derived CD34+ haematopoietic stem and progenitor cells, which are edited using CRISPR at the gamma globin gene (HBG1 and HBG2) promoter sites using a proprietary engineered AsCas12a nuclease, per the company’s website. The first patient had a total haemoglobin level of 16.4 g/dL and 45.4%

Gene Therapy 264

Gene Therapy Drugs Gene Gene Editing 264

Pharmaceutical Technology

MAY 5, 2023

TFF Pharmaceuticals and NIEHS will investigate the TFF-HMW-HA formulations’ therapeutic efficacy and pharmacokinetics using in vitro and in vivo models of respiratory diseases.

Development 162

Development In-Vivo In-Vitro Engineer 162

Pharmaceutical Technology

MAY 17, 2023

Scribe Therapeutics has entered a strategic collaboration with Eli Lilly and Company subsidiary Prevail Therapeutics for accelerating in vivo CRISPR-based therapies to target the causes of serious neurological and neuromuscular diseases. Editorial content is independently produced and follows the highest standards of journalistic integrity.

Genetics 130

Genetics Medicine In-Vivo Gene Therapy 130

Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Our novel editing systems have the potential to precisely modify gene targets for both in vivo and ex vivo therapeutic development.”

Gene Editing 147

Gene Editing Gene In-Vivo Gene Therapy 147

Pharmaceutical Technology

JULY 20, 2022

The alliance plans to leverage the single-cell adeno-associated virus (AAV) engineering (scAAVengr) platform technology of Avista to develop intravitreal AAV capsids for a Roche-defined profile. According to the deal, Roche holds the right to analyse and licence new capsids from Avista. Avista is entitled to receive $7.5m

Gene Therapy 147

Gene Therapy Gene Development In-Vivo 147

pharmaphorum

JANUARY 29, 2021

California-based Sangamo Therapeutics is one such company that believes in the powerful potential of in vivo genome editing and regulation, together known as genome engineering, and has built up a sizable preclinical pipeline of genome regulation treatments for diseases such as Huntington’s disease and Amyotrophic lateral sclerosis (ALS).

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

Pharmaceutical Technology

SEPTEMBER 29, 2022

Under the agreement, Sanofi will receive non-exclusive rights to Scribe’s CRISPR by Design platform of wholly-owned enzymes for developing ex vivo NK cell therapies. The alliance will leverage the CRISPR genome editing technologies of Scribe to facilitate in genetic modification of new natural killer (NK) cell therapeutics for cancer.

Development 130

Development In-Vivo Genomics Genome 130

Pharmaceutical Technology

APRIL 25, 2023

sCFH is an engineered and improved version of complement factor H (CFH) that can fit into adeno-associated virus (AAV) vectors with solid expression and applications confirmed in cultured human cells in vitro, in addition to various preclinical animal models in vivo.

In-Vivo 130

In-Vivo In-Vitro Sales Engineer 130

BioSpace

FEBRUARY 18, 2021

NKarta Therapeutics is using engineered chimeric antigen receptor natural killer T cells (CAR-NKT cells) for allogeneic therapy, showing substantial benefits both in vitro and in vivo, according to James Trager, CSO, speaking Wednesday at the virtual CAR-TCR Summit – Europe.

In-Vivo 74

In-Vivo In-Vitro Engineer 74

Pharmaceutical Technology

SEPTEMBER 14, 2022

As an autologous cell therapy, manufacturing the infusion product is a highly specialised process involving tumour harvest and the extraction and large-scale ex vivo expansion of TILs. The use of engineered immune cytokines that selectively induce the expansion of TILs is being investigated as an alternative to IL-2 stimulation.

Immune Response 246

Immune Response In-Vivo Production Manufacturing 246

Pharmaceutical Technology

FEBRUARY 23, 2023

Innovation S-curve for artificial Intelligence in the pharmaceutical industry In-silico drug discovery is a key innovation area in artificial Intelligence Drug research starts with the discovery stage that involves in vivo and in vitro models to shortlist lead drug compounds.

Drugs 189

Drugs DNA In-Vivo In-Vitro 189

Pharmaceutical Technology

SEPTEMBER 14, 2022

The company is also concluding more than 200 in vivo studies to gain insights and discover new drug candidates that could provide therapeutic radioisotopes for tumour-linked antigens. In addition, our broad discovery engine has generated several novel drug candidates for clinical evaluation in the near future.

In-Vivo 130

In-Vivo Clinical Trials Clinical Trials Engineer 130

pharmaphorum

APRIL 25, 2022

At the moment, it can take weeks to complete the CAR-T process, which includes harvesting of T cells from the patient an shipment to a manufacturing unit, engineering, activation, and expansion of the cells, quality control checks and shipment back to the hospital for re-infusion into the patient.

Engineer 105

Engineer In-Vivo FDA Approval Manufacturing 105

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 DNA Recombinant DNA Technology 130

BioTech 365

OCTOBER 6, 2020

Built by CRISPR leaders including Jennifer Doudna to enable best-in-class in vivo therapeutics that permanently treat the underlying cause of disease Initial $20 million Series A led by Andreessen Horowitz Collaboration with Biogen Inc. focused on development of novel genetic … Continue reading →

Genetics 40

Genetics In-Vivo Engineer Medicine 40

Scienmag

FEBRUARY 10, 2021

Credit: Lei Dong, Nanjing University Scientists from Nanjing University and the University of Macau have devised a new approach to extend the survival of transplanted probiotics in vivo, enhancing the efficacy of cancer chemo-/immunotherapies in mice.

In-Vivo 69

In-Vivo Scientist Engineer Medicine 69

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. Regeneron Pharmaceuticals is the leading patent filer in transgenic murine models.

In-Vivo 130

In-Vivo Antibody Genetics Gene 130

pharmaphorum

FEBRUARY 13, 2022

Quris’ BioAI safety prediction platform – which is based on human tissue samples on chips, nanosensors and machine learning – will be compared to traditional in vitro and in vivo laboratory techniques for spotting toxicity. The post Merck will assess Quris’ AI ‘patient-on-a-chip’ drug safety appeared first on.

Drugs 98

Drugs In-Vivo In-Vitro Pharma Companies 98

XTalks

AUGUST 17, 2020

With several biologics in the pipeline, including one in early stage clinical trials for head and neck cancer, the company is positioned to engineer highly specific biologics against immune targets in the body. Cell-Based Immunotherapy vs. Immune Biologics. Immunotherapy involves leveraging components of the immune system (i.e.

Protein 98

Protein Engineer Immune Response In-Vivo 98

pharmaphorum

FEBRUARY 23, 2021

The inaugural Multi- Functional Cell Therapies Summit gathers the avantgarde to discuss strategies to weaponize cell therapies with antigenic, engineered and combination approaches to create sophisticated, multi-functional systems. NKarta Therapeutics and Iovance Biotherapeutics.

In-Vivo 52

In-Vivo Engineer Development Production 52

XTalks

MAY 22, 2024

He was most recently Vice President of Discovery Research at Codiak Biosciences, where he led the development of a new class of therapeutics based on engineered exosomes. In this episode, Ayesha spoke with John Finn, PhD, Chief Scientific Officer at Tome Biosciences , a company developing programmable gene insertion (PGI) technology.

Gene Editing 52

Gene Editing Life Science Gene Gene Therapy 52

XTalks

MAY 19, 2022

Cardiovascular diseases are the number one cause of death in the world. According to the World Health Organization (WHO), almost 18 million people died from cardiovascular diseases in 2019 , which was 32 percent of all deaths globally. In addition, stents can be used to treat aneurysms and narrowed airways in the lungs.

Development 72

Development Research In-Vivo Engineer 72

pharmaphorum

JANUARY 25, 2022

For example, there is no need for in vivo animal or in vitro pre-clinical or non-clinical studies. The area of digital therapeutics is growing rapidly, and in this piece Ben Hargreaves examines why this is the case, how broadly these therapies can be applied and why investment in the area is mounting. This equated to $21.3

Medicine 111

Medicine In-Vivo In-Vitro Development 111

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

pharmaphorum

OCTOBER 8, 2020

Scribe has designed, engineered and tested thousands of evolved CRISPR enzymes to build an advanced platform for creating breakthrough in vivo treatments,” said Oakes. Benjamin Oakes. At the moment, most CRISPR-based medicines rely on the modification of cells outside the body, that are then used as therapies.

Gene Editing 65

Gene Editing Gene In-Vivo Clinical Trials 65

WCG Clinical

MAY 28, 2024

CAR-T Cells Target Harmful B Cells in Lupus CAR-T cell technology, which uses genetic engineering to direct white blood cells to attack specific molecular targets, was originally proposed for treatment of HIV infection and hematological malignancies. The currently approved CD-19 CAR products are autologous, ex vivo cellular therapies.

In-Vivo 40

In-Vivo Clinical Trials Clinical Trials FDA Approval 40

The Pharma Data

JANUARY 4, 2021

The companies will use ViGeneron’s proprietary vgAAV, novel engineered AAV capsids, to efficiently transduce retinal cells via intravitreal injections. The companies will work together on the in vivo proof of concept (POC). Gene therapy has become a clinical reality. For further information, please visit www.vigeneron.com.

Gene Therapy 52

Gene Therapy Gene Development In-Vivo 52

pharmaphorum

APRIL 29, 2021

To achieve this, the agenda will allow delegates to: Explore the translation of BBB-penetrating antibodies to non-human primates , and new possibilities for in vivo brain imaging of antibody neuropharamacokinetics. A Meeting Point for the Field’s Experts. About Hanson Wade.

Drug Delivery 52

Drug Delivery Gene Therapy In-Vivo Antibody 52

Roots Analysis

DECEMBER 8, 2021

Biopreservation is the exclusive biological approach designated for the preservation of cells, tissues and organs ex-vivo for the purpose of storage, shipment and transportation. Biopreservation media is one of the most crucial entity in conferring effectiveness. F) to 8°C (46.4°F).

In-Vivo 52

In-Vivo Packaging Packaging Biobanking 52

Roots Analysis

SEPTEMBER 18, 2023

Owing to the ability to preserve a wide variety of elements for such a long period of time, including cells, tissues, blood and DNA, cryopreservation has emerged as a key focus area for stakeholders in the biopharmaceutical industry. Therefore, in order to store and preserve such enormous amounts of blood, single-use assemblies are a necessity.

Engineer 52

Engineer Containment In-Vivo Production 52

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. Working alongside KSQ will facilitate smart drug discovery and development of what we hope will be transformative new therapies for patients with intractable forms of cancer.”.

Development 52

Development Research In-Vivo Genomics 52

Delveinsight

FEBRUARY 25, 2021

Electroporation is used for ex vivo delivery of therapies to blood and immune cells. The company’s in vivo therapies reach the eye and central nervous system via adeno-associated viruses, while its liver therapies employ lipid nanoparticles (LNPs). Beam employs three approaches to deliver its genetic medicines to cells.

DNA 52

DNA In-Vivo Genetics Medicine 52

The Pharma Data

JANUARY 11, 2021

Our antibody clones in their original IgG format have shown potent neutralization activity in in vitro assays and, in the case of our lead clone, in an in vivo animal model. Headquartered in Mountain View, California, IGM Biosciences is a clinical-stage biotechnology company focused on creating and developing engineered IgM antibodies.

Antibody 52

Antibody Licensing Licensing Development 52

The Pharma Data

JUNE 17, 2022

Previous research has shown that SARS-CoV-2 spike-specific monoclonal antibodies play a key role in providing in vivo protection and have been used in developing COVID-19 vaccines. A modified version of the Cv2.1169 antibody was also effective at treating SARS-CoV-2 infection in mice and hamsters.

Antibody 52

Antibody In-Vivo In-Vitro Immune Response 52

The Pharma Data

NOVEMBER 5, 2020

PAYSON, UT, November 05, 2020 /24-7PressRelease/ — Dr. Kent R. Van Kampen has been included in Marquis Who’s Who. As in all Marquis Who’s Who biographical volumes, individuals profiled are selected on the basis of current reference value. Van Kampen worked as chief operating officer and chief executive officer of Vaxin, Inc.

Research 52

Research Vaccination Vaccine Doctors 52

XTalks

AUGUST 5, 2020

Single cell sequencing has revolutionized the study of biological tissues and systems at the cellular and molecular level. Recent advances in the technology have allowed for the interrogation of distinct subsets of cell populations within tissues, and associated molecular markers that may function as important disease drivers.

Gene Expression 112

Gene Expression Development RNA Drugs 112

FDA Law Blog

JANUARY 2, 2022

Lenz, Principal Medical Device Regulation Expert — On December 23, 2021, CDRH released as a draft guidance, Assessing the Credibility of Computational Modeling and Simulation in Medical Device Submissions (Draft Guidance). Draft Guidance at 4. Draft Guidance at 8. Draft Guidance at 5.

In-Vivo 52

In-Vivo Development Engineer Clinical Trials 52