Drug Discovery World

APRIL 4, 2023

Read more: New oral option for anaemia of CKD approved in US EA-2353, Endogena Therapeutics The investigation of Endogena Therapeutics’ EA-2353 for the treatment of retinitis pigmentosa (RP) was designated as a Fast Track development programme in February. Here’s a summary. The drug was granted orphan drug designation by the FDA in May 2021.

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FDA Approval In-Vivo Gene Editing Drugs 52

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). The agencies also have different requirements for the selection of healthy donors in the development of allogeneic cell therapies. billion in 2020 to $15.5

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Gene Therapy Marketing Gene DNA 52

The Pharma Data

DECEMBER 1, 2020

NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo , announced today the pricing of an underwritten public offering of 4,794,521 shares of its common stock at a public offering price of $36.50 CAMBRIDGE, Mass., Goldman Sachs & Co.

In-Vivo 40

In-Vivo Containment Genome Genomics 40

XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

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Gene Therapy Gene Trials Clinical Trials 95

Camargo

APRIL 28, 2021

The typical pathway toward initiating pediatric clinical research begins by gathering data in preclinical in vivo studies and in adult healthy volunteers or patients. The next step is the NLMEM approach to develop and optimize the PK model structure, which includes searching for the most statistically and clinically significant covariates.

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Clinical Trials Clinical Trials In-Vivo Drugs 130

The Pharma Data

APRIL 8, 2022

The mere motions of breathing are known to influence vital functions of the lungs, including their development in babies, the production of air-exchange-enhancing fluid on their inner surfaces, and maintenance of healthy tissue structure. a Wyss Technology Development Fellow at the Institute. During his Ph.D.

Immune Response 52

Immune Response In-Vivo Genetic Analysis Research 52

The Pharma Data

OCTOBER 29, 2020

In contrast, OSE Immunotherapeutics is developing OSE-230 as a first-in-class therapeutic agent with the potential to resolve chronic inflammation by driving affected tissues to complete the inflammation program and restore tissue integrity. Most anti-inflammatory agents act using a mechanism that blocks pro-inflammation pathways.

Antibody 52

Antibody In-Vivo Development Clinical Trials 52

Drug Discovery World

OCTOBER 21, 2022

The advance could mean that future development of anti-cancer therapies won’t require the use of animal models. Triple-negative breast cancer cells can develop resistance to the chemotherapy drug doxorubicin. “Our 3D bioprinted breast cancer tumours. 3D bioprinted breast cancer tumours. New target for hard-to-treat breast cancer.

In-Vivo 52

In-Vivo Research Drugs Engineer 52

The Pharma Data

JANUARY 12, 2021

Dr Graham is a medicines development expert and Infectious Diseases Epidemiologist with global Biotech and Pharma R&D experience in Phase I-IV therapeutics as well as in-vivo & in-vitro diagnostics, across many modalities. He has in depth Global Development Expertise (e.g. Dr. Graham earned an M.D.,

Life Science 52

Life Science Antibody In-Vivo In-Vitro 52

Roots Analysis

SEPTEMBER 20, 2023

Further, the development of cell therapy products require a robust population of cells that can be subjected to genetic alterations, without being damaged in this process. In this form of therapy, patients are injected with living and intact human cells that are deemed to be capable of providing therapeutic benefit.

Manufacturing 40

Manufacturing Production Contamination Gene Therapy 40

The Pharma Data

APRIL 22, 2023

“To our knowledge, this study is the first to show in vivo [in a living organism] protection against severe COVID-19 by an AI-designed T-cell vaccine,” said Girish Kirimanjeswara, associate professor of veterinary and biomedical sciences, Penn State. The results were published on April 11 in Frontiers in Immunology.

Vaccination 40

Vaccination Vaccine Protein Antibody 40

XTalks

AUGUST 17, 2020

Immunotherapy continues to be a burgeoning field, with immune-based therapies being among the leading treatments being developed in oncology and autoimmunity. A growing area in the immunotherapy space is the development of biological modulators, which are genetically engineered proteins that target specific components of the immune system.

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Protein Engineer Immune Response In-Vivo 98

The Pharma Data

DECEMBER 13, 2020

Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). The revised plan could support a potential commercial launch of omidubicel in the United States as early as mid-2022.

Licensing 52

Licensing Licensing Clinical Trials Clinical Trials 52

Drug Discovery World

AUGUST 3, 2022

To address these problems, it’s vital to develop delivery systems that enable oligonucleotides to reach their targets: cell-penetrating peptides (CPPs) present a potential solution to this issue. These peptides typically contain up to 30 amino acids and can be cationic, amphipathic or both. How are cell-penetrating peptides used?

Bioavailability 52

Bioavailability In-Vivo RNA In-Vitro 52

The Pharma Data

JANUARY 12, 2021

INCHEON, South Korea–( BUSINESS WIRE )– Celltrion Group today announced top-line results from its randomised, double-blind, and placebo controlled global Phase II/III clinical trial of CT-P59, an anti-COVID-19 monoclonal antibody treatment candidate. days quicker compared to placebo. Patients treated with CT-P59 (40mg/kg) recovered 3.4

Clinical Trials 40

Clinical Trials Clinical Trials Trials In-Vivo 40

The Pharma Data

OCTOBER 21, 2020

We look forward to advancing clinical development of KB-0742 to establish the dose, safety and efficacy of this investigational therapy as a potential treatment for MYC-amplified tumors.”. However, most patients will develop resistance to treatment and progress to CPRC,” said Dr. Koehler. SAN MATEO, Calif., and CAMBRIDGE, Mass.,

In-Vivo 40

In-Vivo In-Vitro Bioavailability Clinical Trials 40

The Pharma Data

DECEMBER 10, 2020

Many people who have been exposed to the “common cold” potentially develop adenovirus immunity: the immune system often attacks and disables these first-generation vaccines before they can activate the immune response to attack the SARS-CoV-2 virus. This blocking of viral replication was observed in both the lung and nasal passages.

Vaccination 52

Vaccination Vaccine Immune Response Protein 52

The Pharma Data

APRIL 22, 2023

Paulson School of Engineering and Applied Sciences (SEAS) has developed a cell therapy as a strong alternative to existing small molecule and protein therapies that leverages myeloid cells, the very type of immune cells that cause the MS-triggering nerve inflammation in patients. who is a Core Faculty member at the Wyss Institute.

Immune Response 40

Immune Response Engineer In-Vivo Containment 40

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. PD-1 has always been a challenge for CAR-T development. “We believe Prof. About PD-1 Check-point inhibition. Dr. Barber is a tumor immunologist.

In-Vivo 40

In-Vivo Development Big Data Immune Response 40

The Pharma Data

DECEMBER 27, 2020

Changes in heparan sulfate (HS) concentration in cerebrospinal fluid (CSF) are being monitored in patients treated with LYS-SAF302 to provide evidence of in vivo biological activity of the drug and demonstrate proof of concept. Average reductions were highly statistically significant. About Lysogene. Sarepta Therapeutics, Inc.

Gene Therapy 52

Gene Therapy Clinical Trials Clinical Trials In-Vivo 52

Drug Discovery World

AUGUST 4, 2022

Developability in the biopharmaceutical industry relates to the difficulty of making early candidate molecules with attractive biological activities into commercialised and marketable therapies. The ‘developability gap’ is the degree of uncertainty when the information needed to make such assessments is lacking at earlier stages of a process.

Development 52

Development Protein Manufacturing Drugs 52

Pharmaceutical Technology

MAY 24, 2023

Every major pharma company is now involved in CGT development which has resulted in the approval of 28 therapies by the FDA thereby making CGT no longer a niche category of therapies. Allogeneic therapies begin with healthy donor samples to develop the eventual therapeutic product which can be administered to multiple patients.

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Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain 246

Drug Discovery World

NOVEMBER 7, 2023

From a personal development perspective, it is an excellent blend of manual technique, high-tech instrumentation, application of basic science, investigation into translational models, and interaction with intelligent and visionary thinkers. We also do our own internal model development and characterisations.

Research 59

Research In-Vivo Gene Therapy Development 59

The Pharma Data

JANUARY 10, 2021

The Company is required to develop and submit to the FDA an improved potency assay prior to enrolling the efficacy phase of the RUBY trial. EDIT-301 is an experimental, ex vivo gene editing cell medicine in development for the treatment of sickle cell disease. Enrolled patients will receive a single administration of EDIT-301.

Medicine 52

Medicine Development Clinical Trials Clinical Trials 52

Roots Analysis

AUGUST 8, 2023

The hydrophobic portion contains two fatty acid chains with 10-24 carbon atoms, while the hydrophilic part primarily consists of phosphoric acid linked to a water-soluble molecule. Introduction to Liposome Liposome was initially identified and described as swollen phospholipid systems, called Banghasomes, by British hematologist Alec D.

Drug Delivery Systems 52

Drug Delivery Systems Drug Delivery Drugs In-Vivo 52

Drug Discovery World

DECEMBER 15, 2022

Possible solutions: A new generation of gene editing tools is being developed to avoid double stranded breaks altogether and to be potentially less destructive than traditional nuclease systems. In vivo approaches involve delivering the gene editing components directly into the patient. In one example, Choi et al.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

Roots Analysis

AUGUST 31, 2023

Further, the transcription factors contain two molecular domains, the DNA binding domain and the activation domain. Moreover, some transcription factors contain additional domains, such as ligand binding domain in order to interact with chemical signals. Figure below presents an overview of the mechanism of action of gene switch.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

Drug Discovery World

AUGUST 15, 2023

Although such electrophysiological systems may be more predictive of potential cardiac safety and efficacy than non-functional binding assays, many apparently attractive new drugs are still being discontinued later in development. Unexpected late-stage failures in drug development are, of course, the costliest.

Drugs 52

Drugs In-Vivo Development Research 52

The Pharma Data

JANUARY 19, 2021

a privately-held neuron regeneration therapeutics company, today announced the publication of results of their novel pharmacotherapy: NNI-362 to allosterically stimulate neural regeneration in human cultures and in vivo models of aging and disease. CLARKSVILLE, Md., 20, 2021 (GLOBE NEWSWIRE) — Neuronascent Inc. ,

In-Vivo 40

In-Vivo Clinical Trials Clinical Trials In-Vitro 40

Drug Discovery World

NOVEMBER 30, 2023

Lightcast is developing a novel, programmable microfluidic platform that allows precise and highly flexible control of individual microdroplets using software-generated light patterns. GSK has developed high-throughput mammalian and E.

Antibody 59

Antibody Protein Engineer In-Vivo 59

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. While disease progression is slower in patients with later-onset SMA, there are significant unmet needs. “We

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

Drug Discovery World

FEBRUARY 1, 2024

Such was the environment in early 2020 when Seth Zost, Pavlo Gilchuck, and several other researchers from Vanderbilt University Medical Center set out to develop a therapeutic antibody against the now infamous virus 1. Time can mean more than money when you’re studying a deadly pathogen.

Antibody 65

Antibody Gene Protein Manufacturing 65

Roots Analysis

MARCH 1, 2023

coli , Salmonella typhi ), as well as its ability to synthesize an essential amino acid required for growth Mutation Assay: Transgenic rodent somatic and germ cell gene mutation assay specifies an in vivo assay that perceives gene mutation causing agents.

Genotoxicity 52

Genotoxicity DNA Genetics Bacteria 52

Drug Discovery World

NOVEMBER 15, 2022

Known for its ability to offer precision and flexibility while expanding the experimental scale, synthetic biology tools can reduce development timelines and costs, and lend a competitive advantage over traditional methods. . Anita Ramanathan outlines four synthetic biology approaches that are improving and accelerating drug discovery. .

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DNA Drugs Antibody 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

The Pharma Data

OCTOBER 26, 2020

Following the unauthorized download of all abstracts on the SITC website, Transgene is communicating the content of the late-breaking poster abstract that will be presented at the SITC 35th Anniversary Annual Meeting (SITC 2020), to be held virtually November 9-14, 2020. Key findings of the trial: .

Vaccination 52

Vaccination Vaccine Trials Containment 52

The Pharma Data

APRIL 27, 2021

Strong increase in Q1 2021 business EPS ( 1) at CER. Q1 2021 sales increase of 2.4% at CER driven by growth drivers Dupixent ® and Vaccines. Specialty Care sales grew 15.3%, due to strong Dupixent ® performance (+45.6% to €1,047 million) and oncology launches. General Medicines core assets grew 4.4%, while GBU sales were down 3.8%.

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Sales Vaccination Vaccine Medicine 52