XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. The webinar included operational strategies for these trials from Laura Omoboni, MS, Executive Director of Clinical Trial Management. Rare diseases can often be progressive, chronic and fatal.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

The Pharma Data

JANUARY 17, 2021

Joe Dupere, CEO of Ixaka, commented: “Ixaka’s broad offering of integrated cell and gene therapy capabilities, encompassing cell-based products and an innovative in vivo gene delivery platform, provides a strong foundation for our ambitions to become a leader in cell and gene therapies. A total of $15.4 A total of $15.4 References 1.

Gene Therapy 40

Gene Therapy Gene In-Vivo Genetics 40

Drug Discovery World

AUGUST 14, 2023

Over the past few decades, the incredible progress in personalised medicine has led to the development of life-saving cell and gene therapies (CGT). For instance, in a trial where patients are selected based on a specific human leukocyte antigen (HLA) type, the FDA requires the use of a fully certified, FDA approved Companion Diagnostic.

Gene Therapy 52

Gene Therapy Marketing Gene DNA 52

Drug Discovery World

JANUARY 23, 2023

Researchers have discovered a clinical drug that sensitises glioblastoma (GBM) cells to radiotherapy and could replace the current standard of care. In addition, they discovered that cladribine, a clinical drug, activates DGKB and inhibits DGAT1. GBM is a WHO grade IV brain tumour with dismal prognosis.

FDA Approval 52

FDA Approval Drugs In-Vivo In-Vitro 52

Pharmaceutical Technology

MAY 24, 2023

By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases.

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Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain 246

Drug Discovery World

JUNE 30, 2023

Research tools, safety testing and regenerative medicines – these endless stem cell applications are powering precision, speed, and new modalities in drug development. Stem cells for use in drug discovery Stem cells are fast becoming an invaluable tool in the drug discovery process. This article is sponsored by Charles River.

Development 52

Development Drugs In-Vitro Gene Therapy 52

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. At the moment, most CRISPR-based medicines rely on the modification of cells outside the body, that are then used as therapies. Benjamin Oakes.

Gene Editing 53

Gene Editing Gene In-Vivo Clinical Trials 53

Drug Discovery World

JULY 22, 2022

For example, in randomised clinical trials of patients with non-small cell lung cancer (NSCLC), pembrolizumab has been shown to be effective in slowing down disease progression and improving survival rates 1. demonstrated the ability to model clinical responses to ACT in autologous HIS mice 4. Jespersen et al.

In-Vivo 52

In-Vivo Engineer In-Vitro Medicine 52

Drug Discovery World

SEPTEMBER 28, 2022

AI-based diagnostic software and clinical trials technology proved to be two of the most attractive health investments, alongside mental health and wellness and medical imaging companies. . But has its application been as revolutionary as industry bods predicted? . The simple answer is no. The unavailability of good data .

Pharma Companies 52

Pharma Companies Drugs In-Vivo Genetics 52

Drug Discovery World

JULY 5, 2023

It’s a brilliantly exciting time where we’re beginning to harvest the fruits of decades’ worth of breakthrough research undertaken by the world’s brightest scientists, and we’re seeing those advances in basic science translated to the clinic, where they’re making a difference in patients’ lives. Mark Garner, PhD.,

Research 98

Research Protein Scientist Immune Response 98

Drug Discovery World

SEPTEMBER 6, 2022

This area of medicine, which turns our own bodies into agents of combat to fight disease, has quickly become one of the most promising fields in treating deadly diseases such as cancer, central nervous system disorders and even musculoskeletal conditions. . billion, compared to $19.9 million price point per dose. Cancer is king .

Gene Therapy 52

Gene Therapy Gene Manufacturing Medicine 52

XTalks

DECEMBER 20, 2023

This includes analyzing how drug combinations may impact individual patients or groups of patients before even entering a clinical trial. With a staggering 90 percent of drugs failing in clinical trials, AI has the potential to help improve these statistics.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

Pharmaceutical Technology

JUNE 15, 2023

Verve Therapeutics and Eli Lilly and Company have entered an exclusive research partnership to advance the former’s preclinical stage in vivo gene editing programme targeting lipoprotein(a) (Lp(a)) to treat atherosclerotic cardiovascular disease (ASCVD). Lilly will provide funding for the Phase I clinical trials.

Gene Editing 130

Gene Editing Gene Gene Therapy In-Vivo 130

XTalks

AUGUST 17, 2020

Cue Biopharma is a Cambridge, Massachusetts-based clinical-stage biotechnology company that is focused on developing injectable immune biologics using its proprietary Immuno-STAT (Selective Targeting and Alteration of T cells) platform. Cell-Based Immunotherapy vs. Immune Biologics. cells, receptors) to treat disease.

Protein 98

Protein Engineer Immune Response In-Vivo 98

The Pharma Data

AUGUST 3, 2021

Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. SVP, Chief Medical Officer, Novartis Gene Therapies. “We

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

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. Gene splicing using nucleases is used to design gene therapeutics for various genetic disorders.

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Gene Splicing Gene Recombinant DNA Technology DNA 130

The Pharma Data

JANUARY 10, 2021

11, 2021 (GLOBE NEWSWIRE) — Editas Medicine, Inc. Food and Drug Administration (FDA) has cleared the initiation of the safety phase of the Company’s EDIT-301 clinical trial, and the Company can begin dosing patients. EDIT-301 is the first experimental medicine in development generated using CRISPR/Cas12a gene editing.

Medicine 52

Medicine Development Clinical Trials Clinical Trials 52

Drug Discovery World

MARCH 20, 2024

Around 30% of drugs show adverse reactions during human clinical trials despite promising pre-clinical studies. Their adaptability for genetic modifications further positions them as an optimal tool for exploring the mechanisms of action of toxic compounds.

Research 59

Research Clinical Trials Clinical Trials In-Vivo 59

Drug Discovery World

DECEMBER 15, 2022

Gene editing tools such as zinc finger nucleases, transcription activator-like effector nucleases (TALEN) and clustered regularly interspaced short palindromic repeat (CRISPR) nucleases have been heralded for their enormous potential to treat diseases and genetic disorders. Early phase clinical trials for gene editing therapies.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

World of DTC Marketing

JANUARY 31, 2021

I have been recruited by these companies who promise huge payouts when they are acquired if their drug shows promise in clinical trials. Ex vivo (where cells are genetically modified outside the body) cell and gene therapies have generated considerable excitement on their potential to cure previously incurable diseases.

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In-Vivo Generic Drugs Gene Therapy Drugs 187

The Pharma Data

MARCH 20, 2023

2,3 These data are among a Zolgensma data set being presented during the 2023 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, which also include, in part, real-world evidence data from the RESTORE registry. Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5

Gene Therapy 40

Gene Therapy Gene In-Vivo Licensing 40

Drug Discovery World

FEBRUARY 15, 2023

Genetically modified T cells stimulated with IL2 are now administered as cell therapy for multiple cancer cell types with beneficial results 3,4. Over 250 clinical trials are currently in progress, and novel immuno-oncological treatments are already proving to be successful. This continues to be the standard of care.

Research 52

Research In-Vitro In-Vivo Development 52

Drug Discovery World

JANUARY 23, 2023

Demonstrating benefits such as fewer side effects and lower cost to enhanced efficacy and accessibility, this emerging therapeutic modality is giving hope to patients and clinicians. The momentum of RNA technologies is apparent in the significant increase in scientific knowledge, IP and deal-making value over recent years.

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RNA Genome Genomics Engineer 98

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.

Vaccination 52

Vaccination Vaccine In-Vivo Genetics 52

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. These efforts, along with many others, are opening doors for new possibilities in translational medicine.

DNA 98

DNA Genome Genomics RNA 98

Roots Analysis

SEPTEMBER 18, 2023

While some biobanks ( especially non-clinical ones ) use mechanical freezers for storage, liquid nitrogen is one of the most reliable cryostorage mediums. million people donate blood annually and on a daily basis, approximately 29,000 units of red blood cells are required.

Engineer 52

Engineer Containment In-Vivo Production 52

The Pharma Data

JULY 8, 2021

The FDA grants Priority Review to applications for medicines that offer significant advantages over available options by demonstrating safety or efficacy improvements, preventing serious conditions or enhancing patient compliance.(1) Results from the NAVIGATOR Phase 3 trial were published in the New England Journal of Medicine in May 2021.

Allergies 52

Allergies Trials In-Vivo Medicine 52

Drug Discovery World

AUGUST 15, 2023

However, development pipelines across multiple therapeutic areas are still not meeting the anticipated clinical outcomes that are needed for bringing new and cost-effective drugs to patients. Such techniques, if routinely deployed in drug discovery, may enable improved decision making in predicting serious adverse cardiovascular events.

Drugs 52

Drugs In-Vivo Development Research 52

Drug Discovery World

JANUARY 23, 2023

In cell therapy, regenerative and adoptive immunotherapies continue to bridge the gap between genetic engineering strategies and their clinical application. This circumvents several technical challenges inherent to the genetic manipulation of differentiated cells in ex vivo culture or intact tissues.

Gene Editing 52

Gene Editing Gene Engineer DNA 52

Drug Discovery World

FEBRUARY 26, 2024

These therapies, which utilise human cells to replace diseased or damaged cells or use engineered cells to act as therapies for a specific disease, have become more common in recent years, with approvals and clinical trials increasing. It’s a laborious process to deliver a single dose of treatment to one patient.

Manufacturing 52

Manufacturing In-Vivo Development Production 52

XTalks

OCTOBER 1, 2020

Dr. Lin received a medical degree from Shanghai Medical University and specialized in hematology after which she began working in the clinical research unit at the university, treating patients with acute leukemia. However, this has since changed, and now, transitioning between academia and industry has become more commonplace. Key Moments.

Scientist 52

Scientist In-Vitro Research Life Science 52

Drug Discovery World

FEBRUARY 22, 2024

According to Medicine Discovery Catapult, these include: “Navigating the complexity of the CNS; understanding the disease pathology, and accessing predictable in vitro and in vivo cell models 2 ”. For example, it can take up to it three years to recruit enough participants for a dementia clinical trial. to £104.7m

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59

Drug Discovery World

MAY 10, 2023

This stage is more highly regulated and consists of both preclinical testing and clinical trials. Until recently, drug carcinogenicity assessment required two different in vivo studies, one of which was typically a two-year lifetime study in rats. Drug discovery and development is an arduous process that can cost upwards of $2.6

Bioassay 52

Bioassay Genotoxicity Drugs In-Vivo 52

Pharmaceutical Technology

APRIL 12, 2023

The Association of the British Pharmaceutical Industry (ABPI) also states that “the use of animals in medical research has played and continues to play an essential role in the development of new medicines and vaccines”. This is to ensure patient safety, as outlined in The Medicines for Human Use (Clinical Trials) Regulations 2004.

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Regulation Vaccination Vaccine Medicine 245