XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. For rare disease studies where there is little or no clinical trial experience, outcome assessments would have to be validated within a clinical development program.

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pharmaphorum

MAY 4, 2022

In testing new treatments, what is promising in animal models may not transfer particularly well to humans, which helps to explain why there is a 90% failure rate during clinical development. An organoid system approach takes self-organising clusters of cells that grow in three dimensions, closely resembling real tissue and organs.

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XTalks

AUGUST 15, 2023

Akeega is approved for use with the corticosteroid prednisone in adults with BRCA1/2 mutations (germline and/or somatic) in whom chemotherapy is not clinically indicated. Niraparib is also sold by GSK under the name Zejula in other indications.

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The Pharma Data

NOVEMBER 9, 2020

(Nasdaq: NVAX), a late-stage biotechnology company developing next-generation vaccines for serious infectious diseases, today announced that the U.S. Currently in late-phase clinical development, NVXCoV2373 is a stable, prefusion protein made using Novavax’ nanoparticle technology and includes its proprietary MatrixM adjuvant.

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The Pharma Data

NOVEMBER 3, 2020

Nasdaq:LOGC) (LogicBio), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, announced today the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its clinical candidate, LB-001 for the treatment methylmalonic acidemia (MMA). LEXINGTON, Mass.,

Clinical Trials 52

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XTalks

NOVEMBER 6, 2020

The Burnaby, BC-based company developed the oral DNA-based vaccine using its proprietary bacTRL Gene Therapy Platform, which uses genetically modified bifidobacteria as carriers of genetic vaccine elements on a DNA plasmid. Related: Red Meat Allergy Test Gets FDA Clearance. “We

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The Pharma Data

JANUARY 19, 2021

Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that it has priced an underwritten public offering of 4,000,000 shares of its common stock at a public offering price of $11.00 CAMBRIDGE, Mass., Forward-Looking Statements.

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The Pharma Data

MARCH 28, 2022

Based on these results, the BIIB078 clinical development program will be discontinued, including its ongoing open-label extension study. “We Vice President and Head of the Neuromuscular Development Unit at Biogen.

Drugs 52

Drugs Genetics RNA Development 52

The Pharma Data

JANUARY 21, 2021

Session: Expanding Targetable Genetic Alterations in NSCLC Mini Oral Session Date and Time: January 31, 2021, 14:20 SGT (January 30, 2021 10:20 p.m. Spectrum Pharmaceuticals is a biopharmaceutical company focused on acquiring, developing, and commercializing novel and targeted oncology therapies.

Clinical Trials 52

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The Pharma Data

OCTOBER 26, 2020

About Prevail Therapeutics Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases.

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Gene Therapy Gene Containment Development 40

The Pharma Data

OCTOBER 27, 2020

Mirati Therapeutics is a San Diego -based late-stage biotechnology company relentlessly focused on translating drug discovery and research into new treatments for patients by advancing and delivering novel therapeutics that target the genetic and immunologic drivers of cancer. Forward-Looking Statements.

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The Pharma Data

DECEMBER 2, 2020

Hemophilia is a genetic hematological rare disease that results in a deficiency of a protein that is required for normal blood clotting—clotting factor VIII in hemophilia A and clotting factor IX in hemophilia B. PFIZER DISCLOSURE NOTICE: The information contained in this release is as of November 23, 2020. About Hemophilia.

Containment 52

Containment Development Clinical Trials Clinical Trials 52

The Pharma Data

SEPTEMBER 8, 2020

Together, these nine companies have collectively developed more than 70 novel vaccines that have helped to eradicate some of the world’s most complex and deadly public health threats, underscoring their experience in clinical development and regulatory rigor, as well as their longstanding commitments to patient safety and public health.

Vaccination 52

Vaccination Vaccine Containment Clinical Trials 52

The Pharma Data

NOVEMBER 20, 2020

Progeria and Progeroid Laminopathies are separate and distinct ultra-rare, genetic, premature aging diseases that accelerate mortality in young patients. While non–progerin producing, these genetic mutations result in disease manifestations with phenotypes that have overlap with, but are distinct from, Progeria.

FDA Approval 52

FDA Approval Genetics Clinical Trials Clinical Trials 52

The Pharma Data

OCTOBER 30, 2020

To develop REGN-COV2, Regeneron scientists evaluated thousands of fully-human antibodies produced by the company’s VelocImmune ® mice, which have been genetically modified to have a human immune system, as well as antibodies identified from humans who have recovered from COVID-19.

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Production Antibody Manufacturing Trials 52

The Pharma Data

SEPTEMBER 8, 2020

Together, these nine companies have collectively developed more than 70 novel vaccines that have helped to eradicate some of the world’s most complex and deadly public health threats, underscoring their experience in clinical development and regulatory rigor, as well as their longstanding commitments to patient safety and public health.

Vaccination 52

Vaccination Vaccine Containment Clinical Trials 52

Drug Discovery World

FEBRUARY 12, 2024

Perhaps the most exciting of these is the ‘organ-on-a-chip’ – a chip the size of a computer memory stick which contains microscopic hollow channels that can be lined with living human cells taken from an organ and through which blood, air and nutrients can be pumped. Clinical Development Success Rates 2006-2015.; 2009;37(SUPPL.

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Medicine Genome Genomics Drugs 59

The Pharma Data

NOVEMBER 4, 2020

Ltd (“Juyou”), a biotechnology company that develops and sells medical and cosmetic skincare products, for the commercialization and development of Pliaglis ® in mainland China (the “License Agreement”).

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Licensing Licensing Dermatology Production 40

pharmaphorum

JANUARY 27, 2023

Minimising any of those delays that happen throughout the clinical trial, or the clinical development is really important and, more and more, it’s about having experts involved in providing advice in that clinical development and clinical trial programme [that] is really important.”

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The Pharma Data

DECEMBER 17, 2020

Senior Vice President and Head of Global Clinical Development at Regeneron and lead author of the publication. patients under an Emergency Use Authorization, and we also continue a robust clinical development program.” “The investigational cocktail is now available to indicated high-risk U.S.

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Antibody Medicine Production Trials 52

The Pharma Data

OCTOBER 27, 2020

NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today announced that the U.S. PHILADELPHIA, Oct. 28, 2020 (GLOBE NEWSWIRE) — Passage Bio, Inc. More information is available at www.passagebio.com. F orward -Looking Statements.

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The Pharma Data

OCTOBER 28, 2020

Senior Vice President and Head of Global Clinical Development at Regeneron. . “We will submit detailed results from this trial for publication in order to share insights with the public health and medical communities,” said David Weinreich, M.D.,

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The Pharma Data

NOVEMBER 11, 2020

Separately, Fast Track Designation facilitates the potential expedited development and review of a drug for the treatment of a serious or life-threatening disease and that has demonstrated the potential to address unmet medical needs. H1 2021: Initiation of Phase 1/2 clinical trial. million as of September 30, 2020.

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Clinical Trials Clinical Trials Trials Development 40

The Pharma Data

DECEMBER 5, 2020

REGN5458 and odronextamab are currently under clinical development, and their safety and efficacy have not been evaluated by any regulatory authority. Additionally, Regeneron bispecifics are manufactured using similar approaches used for human monoclonal antibody medicines, yielding similar properties and pharmacokinetics.

Antibody 40

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The Pharma Data

AUGUST 25, 2020

senior vice president, Global Clinical Development, Hematology, Bristol Myers Squibb. AML is a complex, diverse disease associated with multiple genetic mutations, such as the isocitrate dehydrogenase-2 (IDH2) mutation, and usually worsens quickly and can lead to death if not treated. In certain countries outside the U.S.,

Trials 52

Trials FDA Approval Development Clinical Trials 52

The Pharma Data

DECEMBER 29, 2020

Senior Vice President and Head of Global Clinical Development at Regeneron. . “We appreciate the continued support of patients and investigators around the world who are working to advance Regeneron’s antibody cocktail trials under very challenging circumstances,” said David Weinreich , M.D.,

Antibody 52

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The Pharma Data

SEPTEMBER 20, 2020

Amgen’s clinical development team and clinical investigators, will discuss Phase 1 data being presented on the Company’s investigational KRAS G12C ?inhibitor To learn more about Amgen ‘s innovative pipeline with diverse modalities and genetically validated targets, please visit AmgenOncology.com.

Medicine 52

Medicine Production Sales Clinical Trials 52

XTalks

OCTOBER 26, 2023

She has 23 years of experience working with clients to move vaccine candidates through the clinical development pipeline, including regulatory submission. The Flu versus COVID-19: Virology and Vaccines SARS-CoV-2 versus Influenza Virology Viruses contain genetic material that can either be RNA or DNA.

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The Pharma Data

JANUARY 10, 2021

EDIT-301 is comprised of sickle patient CD34+ cells genetically modified using a highly specific and efficient CRISPR/Cas12a (also known as Cpf1) ribonucleoprotein (RNP) that targets the HBG1 and HBG2 promoters in the beta-globin locus where naturally occurring fetal hemoglobin (HbF) inducing mutations reside.

Medicine 52

Medicine Development Clinical Trials Clinical Trials 52

The Pharma Data

SEPTEMBER 16, 2020

Amgen’s clinical development team and clinical investigators, will discuss Phase 1 data being presented on the Company’s investigational KRAS G12C ?inhibitor To learn more about Amgen’s innovative pipeline with diverse modalities and genetically validated targets, please visit www.AmgenOncology.com.

Production 52

Production Sales Clinical Trials Clinical Trials 52

The Pharma Data

NOVEMBER 5, 2020

Key Pipeline Progress Regeneron has more than 20 product candidates in clinical development, including five marketed products for which it is investigating additional indications. Updates from the clinical pipeline include: Dupixent ® (dupilumab). Non-GAAP net income per share – diluted (1). $. Business Highlights.

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The Pharma Data

OCTOBER 14, 2020

As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases. ELX-02 is in the early stages of clinical development focusing on cystic fibrosis. Forward-Looking Statements.

Protein 52

Protein RNA Production Genetics 52

The Pharma Data

JANUARY 27, 2021

Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders, today announced that the U.S. We are excited to investigate the potential of PBFT02 as a treatment for FTD-GRN as we initiate our clinical development program in the coming months.”.

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Gene Therapy Gene Clinical Trials Clinical Trials 52

The Pharma Data

JANUARY 25, 2021

BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio’s pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development.

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The Pharma Data

OCTOBER 25, 2020

SCD is one of the most prevalent genetic rare diseases in Europe, and despite a clinical understanding of SCD, estimated life expectancy for those affected by the disease is significantly lower than the European average, indicating an urgent need to improve the quality of care for these patients.

Medicine 52

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The Pharma Data

JANUARY 18, 2021

We would like to thank the investigators and the clinical teams for their support and look forward to treating the first patients with this first Invir.IO generated oncolytic virus candidate to enter clinical development” said Dr. Maud Brandely, MD, PhD, Chief Medical Officer of Transgene. Transgene has an ongoing Invir.IO

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The Pharma Data

SEPTEMBER 22, 2020

Trial design and other details are being evaluated and a comprehensive update on the overall Novartis SMA clinical development program will be provided at a future time following further discussions with health authorities. About Spinal Muscular Atrophy SMA is the leading genetic cause of infant death.

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