Drug Discovery World

MARCH 6, 2023

Biopharmaceutical subsidiary Rhythm Pharmaceuticals Netherlands has acquired Xinvento, a Netherlands-based biotech company focused on developing therapies for congenital hyperinsulinism (CHI). Xinvento was founded in 2021 by Claudine van der Sande, an experienced biopharmaceutical leader who previously held positions at Roche and Sanofi.

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Drug Discovery World

FEBRUARY 10, 2023

Breast cancer retained its position as the most-studied disease area in clinical trials in 2022, despite a drop in activity, according to analysis from analytics company Phesi. However, the reduction in breast cancer trials is unexpected, with 113 fewer recruiting trials in 2022 compared with 2021. Download the full report.

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Drug Discovery World

DECEMBER 9, 2022

The ‘Rare Disease Moonshot’, a new initiative to boost research and development into rare and paediatric diseases, has launched at the European Health Summit. . It aims to speed up research into more than 7000 identified rare diseases, of which a staggering 95% have no approved treatment or cure. .

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Drug Discovery World

JULY 11, 2022

David Lewandowski, Business Director of Cell & Gene Therapy at Azenta Life Sciences, explains how efficient sample management can help to work efficiently and bring therapies to market faster. The global cell and gene therapy (CGT) market reached a value of ~$4 billion in 2020 and is expected to grow to ~$34 billion by 2030 1.

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Drug Discovery World

JANUARY 31, 2023

Axicabtagene ciloleucel (Yescarta, Kite) is a CAR-T therapy for adults with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL) who have had two or more lines of systemic therapy. Around 5,500 people are diagnosed with DLBCL each year in England.

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Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. billion, compared to $19.9 Cancer is king .

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Drug Discovery World

MAY 16, 2023

What are the global innovations in cell and gene therapy? What opportunities and challenges are emerging and can therapies get to market faster? Curbishley explains that as the wave of new therapies in development continues to increase, global pressures on manufacturing availability will become more acute.

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PLOS: DNA Science

MARCH 21, 2024

The Food and Drug Administration just announced approval of Lenmeldy (atidarsagene autotemcel), a gene therapy to treat the neurological condition metachromatic leukodystrophy (MLD). The history of gene therapy for MLD is compelling – DNA Science covered it for Rare Disease Day in 2021, here.

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The Pharma Data

MARCH 9, 2021

New data from 10 studies will be presented by Eli Lilly and Company (NYSE: LLY) at the upcoming virtual International Conference on Alzheimer’s & Parkinson Diseases 2021 (AD/PD 2021), March 9-14, 2021. vice president of Alzheimer’s disease development unit, Eli Lilly and Company.

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Drug Target Review

JUNE 19, 2024

The mission of Lineage Cell Therapeutics is to deliver on some of the early promises of cell therapy. Cell therapy as a concept is a wonderful idea, but many of the early efforts never generated the kind of clinical data that gets people excited and leads to new medicines. Hearing aids also have all sorts of deficits.

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Drug Discovery World

SEPTEMBER 17, 2024

Where do you work, and can you tell us what you’re working on at the moment?  I work at Poolbeg Pharma, where we are developing novel treatments for diseases with unmet medical needs and large addressable markets, with programmes in oncology, infectious diseases and endocrinology.

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Drug Target Review

JUNE 3, 2024

How does COUR Pharmaceuticals’ immune-modifying nanoparticle platform differ from traditional approaches to treating immune-mediated diseases? Essentially, our CNPs reprogramme the immune system by restoring balance and returning the body to a homeostatic state, offering a precise approach to treating immune-mediated diseases.

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The Pharma Data

OCTOBER 14, 2021

The results, which demonstrate that patients treated with Biogen’s portfolio of MS therapies mount an effective antibody response to COVID-19 vaccination, are being presented at the 37th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) virtual meeting, October 13-15, 2021.

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The Pharma Data

JULY 8, 2021

Nasdaq:BIIB) today announced it will report second quarter 2021 financial results Thursday, July 22, 2021, before the financial markets open. Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases as well as related therapeutic adjacencies.

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The Pharma Data

APRIL 9, 2021

today announced it will report first quarter 2021 financial results Thursday, April 22, 2021, before the financial markets open. Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases as well as related therapeutic adjacencies.

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The Pharma Data

MARCH 5, 2021

Biogen (Nasdaq: BIIB) today announced it will host webcasts of its pre-recorded presentations and live discussions related to its Alzheimer’s disease investigational therapy, aducanumab, at the upcoming AD/PDTM 2021 Virtual Conference. AD/PD Webcast Schedule Details: Wednesday, March 10, 2021, 6:45 a.m. About Biogen.

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Drug Discovery World

SEPTEMBER 30, 2024

This year, the event will cover therapeutic themes including mechanisms of ageing and cell & gene therapy, along with advances in assay design, applications in high content imaging, target identification and disease modelling. I love to discover and learn how medicines work and to convey this to the broader audience.”

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Drug Discovery World

APRIL 25, 2023

University of California researchers have dosed the second participant in their clinical trial to identify a potential cure for HIV utilising CAR-T cell therapy. The trial is the first-in-human clinical study investigating the duo CAR-T cell therapy for the treatment of HIV. “We In 2021 there were an estimated 1.5

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Drug Discovery World

JULY 23, 2024

Dr Daniel Turner, Chief Scientific Officer, Enhanc3D Genomics, outlines how 3D genomics offers a radically novel approach to address challenges in drug target discovery and how it can affect change in drug target identification, cell therapy and personalised medicine.

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The Pharma Data

JANUARY 25, 2021

25, 2021 (GLOBE NEWSWIRE) — Ambulero, Inc. , a biotechnology company developing cell and gene therapy treatments for patients suffering from vascular disease, today announced that it raised up to $5.5M that will lead clinical testing of a novel gene therapy for a serious vascular disease in Europe.

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The Pharma Data

JUNE 28, 2021

Food and Drug Administration (FDA) granted Breakthrough Therapy designation for donanemab, Eli Lilly and Company’s (NYSE: LLY) investigational antibody therapy for Alzheimer’s disease (AD). Dementia due to Alzheimer’s disease is the most common form of dementia, accounting for 60 to 80 percent of all cases 1.

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Drug Discovery World

JULY 11, 2023

Evotec and BMS aim to identify disease-modifying treatments for a broad range of neurodegenerative diseases. There is a significant unmet medical need for therapies that slow down or reverse disease progression in the field of neurodegenerative diseases.

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Drug Discovery World

MARCH 1, 2024

The trial compared Pepaxto with low-dose dexamethasone to pomalidomide with low-dose dexamethasone in patients with relapsed or refractory (resistant) multiple myeloma following two to four lines of prior therapy and in patients who were resistant to lenalidomide in the last line of therapy.

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Drug Discovery World

NOVEMBER 20, 2023

Biologics represents the fastest-growing sector of the pharmaceutical industry, with hundreds of therapies approved in recent years 3,4. mRNA vaccines and antibody therapies in particular are promising areas that are already having significant impact across the healthcare industry, with enormous potential to address unmet medical needs.

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PLOS: DNA Science

OCTOBER 12, 2023

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.

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The Pharma Data

JULY 29, 2021

econd Quarter 2021 Product Sales Increased 21% Year-Over-Year Primarily Driven by Veklury. Nasdaq: GILD) announced today its results of operations for the second quarter 2021. “We Second Quarter 2021 Financial Results. Total second quarter 2021 revenue of $6.2 As of June 30, 2021, Gilead had $7.4

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The Pharma Data

AUGUST 3, 2021

Revenue in the second quarter of 2021 increased 23 percent, driven by volume growth of 22 percent. – Revenue from all key products grew in the quarter and 2021 year-to-date. . – Revenue from all key products grew in the quarter and 2021 year-to-date. – 2021 EPS guidance updated to be in the range of $6.73

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Drug Discovery World

DECEMBER 7, 2022

In 2021, the global collagen market was valued at $9.75 The collagen trials fell into five main categories: skin disease, wound care, bone disease, osteoarthritis, and rheumatoid arthritis. “As This will be key in ensuring that life-changing therapies make their way to patients faster.”. billion by 2028.

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FDA Law Blog: Drug Discovery

NOVEMBER 2, 2023

Lewis, Senior Regulatory Device & Biologics Expert — On October 20, 2023, FDA announced the availability of the final guidance authored by CBER titled “Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies.” It finalized a draft guidance published in 2022.

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The Pharma Data

JANUARY 11, 2021

Pfizer has started dosing in a phase 3 study evaluating its investigational gene therapy, PF-06939926, in young male patients with Duchenne muscular dystrophy (DMD). The agency previously granted the gene therapy Orphan Drug and Rare Pediatric Disease designations in 2017. Source link.

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Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

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Drug Discovery World

OCTOBER 4, 2022

This year’s event will cover some of the most exciting advancements in areas spanning screening, automation, high content imaging, disease models, cell and gene therapies and how innovation is being driven through partnerships and collaborations. Cell and gene therapies. In 2021, levels of investment hit an all-time high at $22.7

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The Pharma Data

JULY 6, 2021

Sanofi highlights scientific innovations in the field of rare blood disorders at I STH 2021. New research from first-in-class marketed and investigational therapies in hemophilia, immune thrombocytopenia and acquired thrombotic thrombocytopenic purpura will be presented. ePoster Abstract # OC48.2

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The Pharma Data

JANUARY 25, 2021

25, 2021 — Recommendations are presented for the diagnosis and management of von Willebrand disease (VWD) in two clinical guidelines issued by the American Society of Hematology, International Society on Thrombosis and Haemostasis, National Hemophilia Foundation, and World Federation of Hemophilia and published online Jan.

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Drug Target Review

SEPTEMBER 25, 2024

Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.

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The Pharma Data

JANUARY 7, 2021

With eight drugs awaiting regulatory approval and 26 projects in Phase III studies, Sanofi is on the cusp of a transformative year during 2021. However, Sanofi’s programs have significant depth below the waterline that could result in the approval of important medicines to treat difficult diseases. ricochet64/Shutterstock.

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The Pharma Data

APRIL 7, 2021

today announced new data from its expansive neuroscience portfolio will be presented at the 2021 American Academy of Neurology (AAN) Annual Meeting, to be held virtually from April 17-22. . Key AbbVie abstracts and presentation details for the 2021 AAN Annual Meeting program are outlined below. Advanced Parkinson’s Disease.

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