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. The solution?

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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

SEPTEMBER 4, 2024

The drug was approved by the FDA for Expanded Access, allowing trial participants to continue CS1 treatment when no comparable or satisfactory alternative therapy options are available. Topline results will be shared in Q3, 2024.

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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

APRIL 3, 2024

While the age-adjusted overall cancer death rate in the US fell by 33% between 1991 and 2020, cancer remains a significant threat to human health, with more than two million new cases expected to be diagnosed in 2024 and uneven progress across different cancer types 1.

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Drug Target Review

OCTOBER 5, 2023

This study sought to shed light on the safety and effectiveness of aHSCT in routine healthcare settings, moving beyond the confines of clinical trials. The research team identified 231 individuals diagnosed with relapsing-remitting MS, with 174 of them having undergone aHSCT treatment before the year 2020.

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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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FDA Law Blog: Drug Discovery

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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Drug Discovery World

MAY 3, 2024

Importantly, these studies also indicated that emactuzumab has good tolerability and a manageable safety profile. SynOx is initiating a Phase III trial (TANGENT) to assess the efficacy and safety of emactuzumab in patients with locali s ed and diffuse TGCT.

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The Pharma Data

DECEMBER 8, 2020

Blood disease trial participants given Vertex’s and CRISPR Therapeutics’ CRISPR/Cas9-based gene-editing therapy CTX001 demonstrated a “consistent and sustained response” to the treatment, according to new phase 1/2 trial data the companies have announced. 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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The Premier Consulting Blog

MARCH 7, 2024

The FDA’s January 2020 guidance, Chemistry, Manufacturing and Control (CMC) [1] Information for Human Gene Therapy Investigational New Drug Applications (INDs), outlines the analytical methods that define the quality, safety and efficacy of gene therapy therapeutics.

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Drug Discovery World

JANUARY 24, 2023

Virtual clinical trials ‘Virtual’ and ‘technology’ are concepts which are readily exchanged in this industry, but these are more than just buzzwords. The overall virtual clinical trials market is currently valued at $7.8 In early November 2022, two Alzheimer’s drugs were tested head-to-head in a first-ever virtual clinical trial 3.

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The Pharma Data

NOVEMBER 30, 2020

The UK-based RECOVERY trial, which is evaluating multiple treatments for COVID-19, will add the inexpensive anti-inflammatory drug colchicine to the list of therapies being tested in hospitalized patients.

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Drug Target Review

NOVEMBER 7, 2023

Although PROTACs are currently in clinical trials and have shown efficacy in treating cancer , they can only target a protein if it is inside the cell, which is 60 percent of the time. To reach the other 40 percent of those proteins, Stanford ChEM-H researchers pioneered lysosome targeting chimeras (LYTACs) in 2020.

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Drug Discovery World

MAY 29, 2023

“When it comes to published scientific research, the UK is ranked third in the world with nearly 200,000 citable publications in 2020.

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Drug Discovery World

AUGUST 14, 2023

Lung-I Cheng, Vice President and Head of Cell & Gene Therapy Service Line at AmerisourceBergen, and Cori Gorman, Senior Director of CMC and Regulatory Affairs at Biopharma Excellence, offer advice on navigating the different and sometimes contradictory regulatory requirements in the US and EU. billion in 2020 to $15.5 globally.

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Drug Discovery World

JULY 18, 2023

of all new cancer cases in Europe in 2020, with around 136,000 new cases diagnosed. The MAA is based on results from the Phase III SPOTLIGHT and GLOW clinical trials. In both SPOTLIGHT and GLOW, approximately 38% of patients screened for the trials had tumours that were CLDN18.2-positive Gastric cancer accounted for 3.1%

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The Pharma Data

DECEMBER 21, 2020

FDA Approves First Oral Hormone Therapy for Advanced Prostate Cancer. 21, 2020 — Orgovyx (relugolix) is now approved to treat advanced prostate cancer and is the first oral hormone therapy approved for this indication, the U.S. © 2020 HealthDay. Posted: December 2020. MONDAY, Dec. More Information.

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The Pharma Data

DECEMBER 30, 2020

Some people in clinical trials of the Oxford-AstraZeneca vaccine were given the two doses several months apart. Using the contaminated mouthwash can result in “oral and, potentially, systemic infections requiring antibacterial therapy. © 2020 HealthDay. Posted: December 2020. All rights reserved. Source link.

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The Pharma Data

SEPTEMBER 1, 2020

Daiichi Sankyo has unveiled promising new benefit for Nilemdo (bempedoic acid) at the ESC Congress 2020 over the weekend for the management of low-density lipoprotein cholesterol (LDL-C) when added to other standards of care. In this extension trial, 970 participants received Nilemdo and 492 received placebo.

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The Pharma Data

DECEMBER 8, 2020

The FDA has issued a clinical hold on Bellicum Pharmaceuticals for its ongoing phase 1/2 trial evaluating CAR-T therapy BPX-601 in patients who received prior treatment for metastatic pancreatic or prostate cancer, following the death of a trial participant. The Houston, Tex., The Houston, Tex., Source link.

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The Pharma Data

DECEMBER 11, 2020

11, 2020 — Results from a long-term study of a gene therapy technique to prevent inherited mitochondrial disease show promise, researchers say. The therapy under investigation replaces disease-causing mutations in a mother’s egg with donor mitochondria. FRIDAY, Dec. in the small intestine.

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New Drug Approvals

SEPTEMBER 8, 2024

2222514-07-8 ] Palopegteriparatide , sold under the brand name Yorvipath , is a hormone replacement therapy used for the treatment of hypoparathyroidism. [1] 5] History The effectiveness of palopegteriparatide was evaluated in a 26-week, randomized, double-blind, placebo-controlled trial that enrolled 82 adults with hypoparathyroidism. [5]

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The Pharma Data

NOVEMBER 4, 2020

5, 2020 11:00 UTC. NASDAQ: CNCE) today reported financial results for the third quarter of 2020. Our team has diligently and effectively executed on our plan, and I’m pleased that we have achieved our stated 2020 pipeline milestones. Initiated CTP-543 THRIVE-AA1 Phase 3 Trial in November 2020.

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The Pharma Data

SEPTEMBER 10, 2020

(NYSE:PFE) announced today that more than 50 abstracts representing data from nine approved and investigational Pfizer medicines, including several biomarker-driven and immuno-therapies, will be presented at the European Society for Medical Oncology (ESMO) Virtual Congress 2020 being held from September 19-21, 2020. Mayer E. .

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Drug Discovery World

FEBRUARY 28, 2024

Using IgE to treat cancer In the UK, a Cancer Research UK-funded clinical trial has shown, for the first time, that a new class of antibody could benefit cancer patients whose existing treatments have stopped working. Cancer shredding could hold potential not only for glioblastoma, but possibly for other hypermutated tumours.”

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Drug Discovery World

OCTOBER 4, 2022

The approval is the result of a long advocacy campaign to convince the FDA to approve the treatment prior to completion of an ongoing Phase III trial. . “We In June 2016, the Association provided Amylyx, the producer of AMX0035, with a $750,000 grant for a clinical trial pilot.

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The Pharma Data

DECEMBER 22, 2020

23, 2020 /PRNewswire/ — Rakuten Medical, Inc. Approximately 74 patients are planned for this trial in Head and Neck Squamous Cell Carcinoma (HNSCC) and Cutaneous Squamous Cell Carcinoma (CuSCC). Outside of Japan , Illuminox therapies have not yet been approved as safe or effective by any regulatory authority.

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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

OCTOBER 26, 2020

27, 2020 10:45 UTC. –( BUSINESS WIRE )– Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced financial results for the third quarter of 2020. 2020. for the third quarter of 2020. 2020. for the third quarter of 2020. .

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The Pharma Data

DECEMBER 22, 2020

Tagrisso Approved as Adjuvant Therapy for NSCLC With EGFR Mutations. 22, 2020 — Tagrisso ( osimertinib ) received approval as an adjuvant treatment for patients with non-small cell lung cancer with epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R mutations, the U.S. © 2020 HealthDay. Professional.

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The Pharma Data

DECEMBER 9, 2020

10, 2020 04:32 UTC. NASDAQ: RCKT) (“Rocket”), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the pricing of an upsized underwritten public offering of 4,642,858 shares of its common stock at a public offering price of $56.00

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The Pharma Data

OCTOBER 27, 2020

28, 2020 05:00 UTC. Dry AMD is one of the leading causes of vision loss globally and gene therapies hold tremendous promise for this disease. Delivering gene therapy directly to the site of disease through a subretinal injection is a proven approach; however, the current method requires a vitrectomy. About FOCUS.

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The Pharma Data

JUNE 28, 2021

Parkinson’s disease is most common neurodegenerative movement disorder / It impacts more than 10 million people worldwide/ No function-restoring therapy is currently available / Bayer is pursuing a two-pronged approach to deliver transformative therapies with one cell and one gene therapy candidate in clinical trials.

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The Pharma Data

NOVEMBER 18, 2020

19, 2020 /PRNewswire/ — OBI Pharma, Inc. “Based upon our anti-Globo H targeted approaches in cancers of high unmet needs, OBI Pharma is proud to have presentations on the progress of our trial presented at ESMO-Asia 2020 for our novel therapeutic cancer vaccine, OBI-833.” TAIPEI, Taiwan , Nov. About OBI Pharma.

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The Pharma Data

AUGUST 7, 2020

As the pharma industry stands firm in its commitment to advance the sector to fight Covid-19, news has emerged from the European Commission who intend to streamline the development of therapies using genetically modified organisms to treat Covid-19. Regenerative medicine and advanced therapies thriving despite Covid-19 disruption.

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