Pharmaceutical Technology

MAY 24, 2023

Autologous therapies are characterized as a circular supply chain; the patient’s own sample starts the supply chain, and it is then subjected to ex-vivo modifications with the resulting therapy administered back to that same patient. What are the general differences in the supply chain of CGT vs. traditional clinical trials?

Clinical Trials 246

Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain 246

Scienmag

NOVEMBER 16, 2020

Wnt-regulated lncRNA discovery enhanced by in vivo identification and CRISPRi functional validation. Singapore scientists uncover potential role of long non-coding RNAs in pancreatic cancer Credit: From Figure 4 in Liu, S., Harmston, N., Glaser, T.L. Genome Med 12, 89 (2020).

In-Vivo 53

In-Vivo RNA DNA Genome 53

Drug Patent Watch

JANUARY 14, 2025

”[2] This study not only addressed the ATA’s concerns but also highlighted the power of real-world evidence in generic drug development and regulation. .”[2] ”[2] This study not only addressed the ATA’s concerns but also highlighted the power of real-world evidence in generic drug development and regulation.

Generic Drugs 87

Generic Drugs Development Drugs Manufacturing 87

pharmaphorum

AUGUST 10, 2020

The filing with the FDA follows approval by several regulators across the world, including in the European Union and Japan. Originally intended as a treatment for Ebola virus, Veklury is a nucleotide analogue with broad-spectrum antiviral activity both in vitro and in vivo in animal models against several emerging viruses.

Drugs 99

Drugs In-Vivo In-Vitro Contract Manufacturing 99

FDA Law Blog

MARCH 19, 2024

using phantoms, ex vivo animal tissue models, and/or in vivo animal testing), computationally, and/or clinically. When in vivo animal testing is needed, tests should follow good laboratory practices and the animal model should be representative of the intended clinical application. By Adrienne R.

In-Vivo 59

In-Vivo Marketing Regulation Development 59

BioTech 365

NOVEMBER 11, 2021

Gain Therapeutics Presents Parkinson’s Disease Preclinical Study Results at Society for Neuroscience Annual Meeting Gain Therapeutics Presents Parkinson’s Disease Preclinical Study Results at Society for Neuroscience Annual Meeting Brain-penetrant structurally targeted allosteric regulators for glucocerebrosidase (GCase) show promising in vivo … (..)

In-Vivo 40

In-Vivo Regulation 40

Pharmaceutical Technology

SEPTEMBER 10, 2008

IS: Our genotoxicity testing includes the core battery of the tests requested by EMEA/ICH including mutagenicity in vitro, chromosomal aberration test in vitro and micronucleus test in vivo. Natalie Coomber: Can you provide a quick background on the history of Biotest and the main work it is currently undertaking?

In-Vitro 100

In-Vitro Genotoxicity In-Vivo Development 100

pharmaphorum

JULY 22, 2021

All of our processes, and also those of the entire healthcare ecosystem, including regulators, had to be accelerated. In just over a year, Celltrion Healthcare says it has identified and delivered a potential treatment for COVID-19. pharmaphorum spoke to Kwon Ki-sung, Head of Celltrion’s R&D unit, to learn more.

Antibody 105

Antibody Clinical Trials Clinical Trials Trials 105

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.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

Roots Analysis

AUGUST 31, 2023

Type of Gene Regulation Based on the type of transcription factor ( activators / repressors ), gene regulation has been categorized into positive and negative gene regulation. Negative Gene Regulation: Repressor is a protein molecule that initiates negative regulation.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

The Pharma Data

DECEMBER 9, 2020

The Chinese biopharmaceutical industry is growing in leaps and bounds, but there is still a huge unmet need when it comes to getting patients access to the breakthrough therapeutic modalities and platforms like RNAi, cell and gene therapy and others. A solution could be at hand with Overland Pharmaceuticals.

In-Vivo 52

In-Vivo In-Vitro Antibody Development 52

Roots Analysis

DECEMBER 22, 2021

In recent years, Treg based therapies have become a research hotspot in the field of immunotherapy due to their ability to prevent / delay graft rejection and control autoimmune responses generated post adoptive transfer in vivo. Mechanism of Tregs. For further information on this domain, check out the report –. Our Social Media Platform.

Immune Response 52

Immune Response Research In-Vivo Marketing 52

pharmaphorum

JANUARY 25, 2023

Digital therapeutics company Woebot Health has announced enrolment of the first patient in a pivotal clinical trial to evaluate the safety and efficacy of WB001, an investigational therapeutic for postpartum depression (PPD), granted Breakthrough Device designation by the US Food and Drug Administration (FDA) in 2021.

In-Vivo 105

In-Vivo Clinical Trials Clinical Trials Trials 105

WCG Clinical

MAY 28, 2024

When properly regulated these cellular features—proliferative capacity and receptor-directed specificity—largely account for the effectiveness of our adaptive immune system in protecting us from infectious disease. 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

Roots Analysis

JANUARY 27, 2025

In order to manufacture GMP regulated cell therapies, it is necessary to assess each manufacturing step at regular intervals. In this form of therapy, patients are injected with living and intact human cells that are deemed to be capable of providing therapeutic benefit. This figure presents the cell therapy manufacturing steps.

Manufacturing 40

Manufacturing Contamination Production Gene Therapy 40

Pharma in Brief

JUNE 14, 2022

The Federal Court has allowed an infringement action concerning a macitentan product under the Patented Medicines (Notice of Compliance) Regulations (the Regulations ). The Court dismissed allegations that the asserted patent claims were invalid for obviousness, lack of utility, overbreadth, and insufficiency of disclosure.

Containment 52

Containment In-Vivo Regulation Production 52

pharmaphorum

DECEMBER 6, 2020

CTX001 is an ‘ex vivo’ application of gene-editing, in which the technology is used to modify a patient’s own cells outside the body. Similarly, three SCD patients suffered none of the characteristic painful attacks – known as vaso-occlusive crises (VOCs) – in the three to 15-month follow-up period after the treatment.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

XTalks

SEPTEMBER 20, 2024

Such challenges highlight a significant unmet need and call for further advancements in the field. Vittoria Biotherapeutics is a clinical-stage cell therapy company that incorporates the latest advancements in cell therapy and gene editing with a unique approach that potentially overcomes some of the issues with this therapeutic modality.

Gene Editing 52

Gene Editing Manufacturing Gene In-Vivo 52

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). By Adrienne R. Draft Guidance at 4. Draft Guidance at 8. Draft Guidance at 5.

In-Vivo 52

In-Vivo Development Engineer Clinical Trials 52

Delveinsight

FEBRUARY 11, 2021

Late in 2019, a Fabry patient in phase 2 clinical trial of ex vivo lentiviral gene therapy AVR-RD-01 became the first patient dosed using Plato. The study will be launched by Verily’s Project Baseline testing program and intends to collect biological information and real-world data in the weeks soon after a person tests positive for COVID-19.

Gene Therapy 52

Gene Therapy Gene Immune Response In-Vivo 52

Delveinsight

JANUARY 18, 2021

For instance, GTX-102 , an antisense therapy silencing the gene expression by binding to the RNA molecule behind the regulation of its expression, has also secured USFDA Fast Track Designation. Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system. GeneTx and Ultragenyx are co-developing the therapy.

Marketing 52

Marketing Gene Drugs Protein 52

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). By Adrienne R. Draft Guidance at 4. Draft Guidance at 8. Draft Guidance at 5.

In-Vivo 40

In-Vivo Regulation Development Engineer 40

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.

Life Science 52

Life Science Antibody In-Vivo In-Vitro 52

Pharma in Brief

FEBRUARY 3, 2021

The Court’s decision concerned the claims of two patents asserted against each of the Defendants in infringement actions under the Patented Medicines (Notice of Compliance) Regulations ( Regulations ): Canadian Patent No. Background. ELIQUIS TM (an blood thinner) is marketed in Canada by Bristol-Myers Squibb Canada Co.

In-Vivo 52

In-Vivo Regulation Compliance Life Science 52

The Pharma Data

JANUARY 11, 2021

The first co-published, peer-reviewed research paper between a commercial MPS provider and a regulator, demonstrates that data derived using CN Bio’s proprietary PhysioMimix system is appropriate for use in drug safety and metabolism applications, evidencing its enhanced performance versus standard techniques.

In-Vivo 40

In-Vivo Drugs Clinical Trials Clinical Trials 40

The Pharma Data

NOVEMBER 10, 2020

Hydrogen-Oxygen Inhalation for Treatment of COVID-19, a monograph concluding China’s experience in the fight against COVID-19, reports that Hydrogen Oxygen Generator with Nebulizer is effective in treating patients with COVID-19 coronavirus. . . SHANGHAI , Nov. SHANGHAI , Nov.

Medicine 52

Medicine In-Vivo Clinical Trials Clinical Trials 52

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 Gene 52

XTalks

FEBRUARY 10, 2023

According to a recent review published in Nature , as of April 2022 almost 1,800 active cell therapy clinical trials were listed in ClinicalTrials.gov, a 33 percent increase from 2021. The rapidly expanding landscape of cell therapy trials has put an operational strain on many clinical sites due to limited resources and infrastructure. “In

Clinical Trials 52

Clinical Trials Clinical Trials Trials Gene Therapy 52

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

DECEMBER 23, 2020

The European Union (EU) authorized ViiV Healthcare ’s Vocabria (cabotegravir injection and tablets) in conjunction with Janssen’s Edurant (rilpivirine tablets) for the treatment of HIV-1 infection in adults who are virologically suppressed. Elsewhere around the world: Avacta Group – U.K. It is a division of Gamma Biosciences.

Drugs 52

Drugs Licensing Licensing Vaccination 52

FDA Law Blog

OCTOBER 10, 2023

By Philip Won & Véronique Li, Senior Medical Device Regulation Expert — As we recently blogged , FDA released three draft guidance documents to help enhance the predictability, consistency, and transparency of the 510(k) program. One of these documents focuses on “ Evidentiary Expectations for 510(k) Implant Devices.”

In-Vivo 45

In-Vivo Manufacturing Engineer Genotoxicity 45

XTalks

AUGUST 12, 2020

Despite the effectiveness of anti-retroviral therapy (ART) in significantly improving health, quality of life and reducing mortality among HIV-positive individuals, new research shows that persistent infections may be due to the ability of the virus to take refuge in distinct subsets of immune T cells.

RNA 52

RNA Protein DNA In-Vivo 52

FDA Law Blog

OCTOBER 11, 2022

Moreover, the draft guidance is the first time FDA tried to explicitly describe its position on such a key phrase (“confirmatory evidence”) that was made a part of the plain language of the statutory standard for drug approval in 1997, a 22-year interval of near public silence.

Clinical Trials 61

Clinical Trials Clinical Trials Drugs Trials 61

The Pharma Data

JANUARY 27, 2021

Hayden, CEO of Prilenia and world-renowned scientist in Huntington’s Disease research , commented: “The design of the PROOF-HD study is based on strong scientific and clinical data, including in vivo target engagement for the selected dose, prior clinical efficacy results and extensive long-term safety data in our target population.

Clinical Trials 40

Clinical Trials Clinical Trials Trials In-Vivo 40

Roots Analysis

JANUARY 14, 2024

Like all drugs, biologics are regulated by the FDA. Notably, biologics have higher success rates than conventional small-molecule drugs owing to lesser off-target toxicity of biologics, which is one of the key reasons of failures in small molecule drugs development.

Contract Manufacturing 40

Contract Manufacturing Protein Manufacturing Production 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. 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

XTalks

OCTOBER 1, 2020

Leaving academia for industry is a route that academic scientists and professors did not traditionally pursue in the past due to perceived differences in skillsets, experiences and even associated stigmas. 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