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.

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XTalks

AUGUST 17, 2020

This typically involves taking T cells from the body, engineering chimeric receptor antigens (CAR) and inserting them into the cells, expanding the cells ex vivo and injecting them back into the body. Cell-Based Immunotherapy vs. Immune Biologics. Immunotherapy involves leveraging components of the immune system (i.e.

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Roots Analysis

SEPTEMBER 18, 2023

Due to the infrastructure available for processing, freezing and storing of these container systems, blood bags are the preferred container for cell therapy biopreservation. While some biobanks ( especially non-clinical ones ) use mechanical freezers for storage, liquid nitrogen is one of the most reliable cryostorage mediums.

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Roots Analysis

JANUARY 27, 2025

Such procedures require sterile and higher-grade clean rooms, along with regular checks on containment levels. The American Society of Gene and Cell Therapy (ASGCT) defines cell therapy as a therapeutic modality that involves the administration of either normal or modified cells to patients for the treatment of various diseases.

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The Pharma Data

JANUARY 10, 2021

EDIT-301 is an experimental, ex vivo gene editing cell medicine in development for the treatment of sickle cell disease. 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. Enrolled patients will receive a single administration of EDIT-301.

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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.

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Genotoxicity DNA Genetics Bacteria 52

The Pharma Data

APRIL 8, 2022

Every breath stretches the lungs’ tissues with each inhale and relaxes them with each exhale. 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. During his Ph.D.

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Immune Response In-Vivo Genetic Analysis 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. , About NNI-362.

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The Pharma Data

OCTOBER 29, 2020

The oral presentation entitled “ Agonist anti-ChemR23 mAb blunts tissue neutrophil accumulation and triggers chronic colitis inflammation resolution ” shows efficacy results for OSE-230 in chronic inflammatory preclinical models and ex vivo human models. 7 therapies.

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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.

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Drug Delivery Systems Drug Delivery Drugs In-Vivo 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. Upon binding, the antibodies block the spike protein from interacting with ACE2 and thereby prevent virus-induced cell-killing, also known as cytopathic effect.

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The Pharma Data

DECEMBER 27, 2020

SOUTH SAN FRANCISCO, Calif. and TAIPEI, Taiwan, Dec. TLC599 is a non-opioid, proprietary BioSeizer ® sustained release formulation of dexamethasone sodium phosphate (DSP) intended to manage OA pain for up to six months. According to the US Department of Health and Human Services, 10.3 About TLC599.

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WCG Clinical

AUGUST 30, 2024

Data from the Alzheimer’s Association estimates that approximately 5.4 million Americans are living with Alzheimer’s disease today at an estimated annual cost of $183 billion dollars. Of Americans aged 65 and older, 1 in 8 has Alzheimer’s disease and nearly half of people aged 85 and older have the disease.

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Pharma Marketing Network

JANUARY 20, 2021

Assuming these findings are replicated in clinical trials, this vaccine, with its more-appealing route of administration and convenient (refrigerator) storage requirements could make an important contribution to containing the disease.

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Roots Analysis

AUGUST 23, 2024

Most current flu shots contain live but weakened viruses; adenoviral vector-vaccines like AstraZeneca’s encode Sars-Cov-2 onto another virus which itself might cause illness; and subunit-vaccines contain lab-created pieces of viral coating. Ribosomes are cellular machines that read mRNA sequences and produce proteins.

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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.

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Gene Therapy Clinical Trials Clinical Trials In-Vivo 40

The Pharma Data

OCTOBER 21, 2020

An in vivo efficacy study examined the oral administration of KB-0742 in mice that had been engrafted with the human CRPC cell line and compared tumor growth to standard docetaxel chemotherapy (intraperitoneally at 15 mg/kg, once weekly), or escalating doses of KB-0742 (orally at 3, 10, and 30 mg/kg, once daily) over 21 days.

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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.

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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.

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The Pharma Data

APRIL 22, 2023

These backpacks are loaded with anti-inflammatory molecules that direct the carrier cells’ differentiation into anti-inflammatory cells in vivo. The Atlas of MS reported in 2020 that someone is diagnosed with MS every five minutes around the world, adding to about 2.8 million individuals that currently have to live with the disease.

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Roots Analysis

SEPTEMBER 20, 2023

Cell therapies are based on the premise that the patient’s own cells ( autologous ), or those from a healthy donor ( allogeneic ), can be genetically re-programmed to combat various diseases. In this form of therapy, patients are injected with living and intact human cells that are deemed to be capable of providing therapeutic benefit.

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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.

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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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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

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Clinical Trials Clinical Trials Gene Therapy Gene 52

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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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: .

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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

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Clinical Trials Clinical Trials Trials In-Vivo 40

The Pharma Data

DECEMBER 10, 2020

.–( BUSINESS WIRE )– ImmunityBio , a privately-held immunotherapy company, today announced its COVID-19 vaccine candidate protected nasal and lung airways of non-human primates against coronavirus (SARS-CoV-2) in a challenge study. This blocking of viral replication was observed in both the lung and nasal passages.

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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. THIS ANNOUNCEMENT CONTAINS INSIDE INFORMATION FOR THE PURPOSES OF ARTICLE 7 OF REGULATION (EU) NO 596/2014.

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The Pharma Data

DECEMBER 13, 2020

The company believes the clinical comparability requirement will be met if the time to neutrophil engraftment in patients from the company’s ongoing expanded access program (EAP) using omidubicel produced at Gamida Cell’s planned commercial manufacturing sites is consistent with the results achieved in the Phase 3 clinical trial. About Omidubicel.

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Licensing Licensing Clinical Trials Clinical Trials 52

Pharma in Brief

JUNE 14, 2022

OPSUMIT® is a tablet containing macitentan. The Court dismissed allegations that the asserted patent claims were invalid for obviousness, lack of utility, overbreadth, and insufficiency of disclosure. Background. Janssen Inc. Janssen ) markets OPSUMIT® in Canada for the treatment of pulmonary arterial hypertension ( PAH ). Sandoz Canada Inc.

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The Pharma Data

DECEMBER 20, 2020

. PARTNERSHIP LEVERAGES COMPLEMENTARY STRENGTHS OF IN SILICO, IN VITRO AND IN VIVO ANTIBODY DISCOVERY IN A RANGE OF INDICATIONS. Alloy’s first platform, the ATX-Gx(TM) mouse platform, is a suite of transgenic mice designed for best-in-class in vivo discovery of fully human monoclonal antibodies.

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FDA Law Blog

DECEMBER 6, 2024

Because this guidance covers such a breadth of information, for Part 1 of our coverage, we will focus on the non-CMC topics and summarize our top takeaways from each section, something of a Cliffs Notes for the Cliffs Notes. Section #1: FDA Interactions Given the wide range of sponsors (i.e.,

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