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Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Read it on our website here. Illustration by David S. Fast forward to 2023.
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.
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. She passed away in 2013.
These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. Patients and caregivers also assess the benefits offered by different therapies, weighing the progression-free survival with their off-target effects. 3D rendering of Antibody Drug Conjugate Molecules.
Over the past 25 years, T-cell therapies have gained significant ground in the treatment of cancer. Preclinical research on γδ T cells has made great strides since the cells were first identified in the 1980s, with γδ T-cell therapies from several companies, including IN8bio, now in or nearing clinical trials for various cancers.
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.
Results from the Phase III EMPEROR-Preserved trial were presented today at the European Society of Cardiology Congress 2021 2 and published in The New England Journal of Medicine 1. 1 Trial participants were randomly assigned to empagliflozin 10 mg (n=2,997) or placebo (n=2,991) once daily.
In addition to this, I also chaired national trials evaluating an anti-GD2 antibody in pediatric osteosarcoma. Working with one of the largest cooperative groups in pediatrics, the Children’s Oncology Group , we ran quick signal-seeking trials to determine efficacy.
million people worldwide were living with HIV in 2021. Several agents are in clinical trials, including apricitabine, racivir, elvucitabine, doravirine, dapivirine, and elsulfavirine. Abstract Human immunodeficiency virus (HIV) causes acquired immunodeficiency syndrome (AIDS), a lethal disease that is prevalent worldwide.
Sponsors using master protocols to test new drugs and therapies for treating or preventing COVID-19 should base their analyses on comparisons between control arm participants who were concurrently randomized, the FDA advised in a final guidance released yesterday. Read the guidance here: www.fdanews.com/05-17-21-COVID-19.pdf.
Data demonstrate potential of Blenrep (belantamab mafodotin-blmf) in combination with standard of care therapies in patients with newly diagnosed and relapsed/refractory multiple myeloma. Full list of belantamab mafodotin DREAMM trials and analyses.
Moreover, the probability of success for a drug candidate entering clinical trials is only around 10%, highlighting the substantial risks involved. reported in 2021. In 2021, an estimated 537 million adults were living with diabetes, a number projected to reach 643 million by 2030. billion in 2021, is projected to reach 231.7
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
Research in gene therapies and genetically engineered drugs and vaccines are growing exponentially, and will only continue to become more popular. The accelerating gene therapy market is expected to grow globally by 16.6% An IRB tends to get the most attention since all human trials need IRB approval. between 2020-2027.
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.
But immunotherapy was not yet widely used and had not been applied clinically to Merkel cell carcinoma, so she received traditional chemotherapy and radiation therapy, suffering life-threatening complications along the way. By then, a few cancer immunotherapies had entered clinical trials and only a handful had been approved by the FDA.
Presentations: 535P, 536P, 534P, 104P, 1534P, 444P, 630P, LBA26, 623P, 603P, 593P, 594P, 1309P, 606P, 590P, 826O, 530P, LBA58, 1520O, 48P, 508TiP, 505TiP, 509TiP, 1438TiP Bayer will present new data from its established portfolio of approved oncology therapies at the upcoming ESMO Congress 2021 from September 16-21, 2021.
She is scheduled to be the 2nd patient in the world to receive ASO therapy for KAND. Timeline: December 2021 – We met with Dr Chung in NYC and discussed and applied to n-Lorem. March 2023 – We met with n-Lorem and found out Susannah’s ASO (1st KAND patient to receive ASO therapy for KAND) would work for Sloane.
to $6.14; Raises 2021 Adjusted Diluted EPS Guidance Range from $12.37 Based upon the momentum of our business, we are raising our full year 2021 EPS guidance and believe AbbVie is very well positioned for the long term.” ” Second-Quarter Results. Worldwide net revenues were $13.959 billion, an increase of 33.9
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
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.
Additionally, in 2021, a literature review estimated the average capitalized research and development (R&D) costs per new cancer medicine at between $944 million and $4.54 Innovation Organizations conducting oncology clinical trials face challenges distinct from the rest of the research community.
Below is a chart that looks at 3 numbers – the number of AdComms; the number of new molecular entities approved each year, and the number of drugs that had breakthrough therapy designation. That said, BT would be a lagging indicator since it can be granted so early in the development process. Fewer meetings – more approvals.
These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline. Additional trials (e.g.,
Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for empagliflozin as an investigational treatment for adults with heart failure with preserved ejection fraction (HFpEF), Boehringer Ingelheim and Eli Lilly and Company (NYSE: LLY) announced. o Completion: 2021. o Number of patients: 3,730.
However, this dropped under 10% in 2021. From Early Transfusion to Component Therapy The earliest documented attempts at blood transfusions involved the transfer of blood from animals to humans in the 17 th century. However, the subsequent death of another patient thrust Denys into a contentious trial. Subscribe to Asimov Press.
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
Advancing reduction of drug use as an endpoint in addiction treatment trials astewart Thu, 03/06/2025 - 09:59 Nora's Blog March 18, 2025 Image Getty Images/ SolStock This blog was also published in the American Society of Addiction Medicine (ASAM) Weekly on March 18, 2025.&
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.
Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for empagliflozin as an investigational treatment for adults with coronary failure with preserved ejection fraction (HFpEF), Boehringer Ingelheim and Eli Lilly and Company (NYSE: LLY) announced. 2,3 a number one explanation for hospitalization within the U.S.
In the first half of 2021, Boehringer Ingelheim has supplied more humans and animals worldwide with innovative medicines than ever before. A recent trial showed a significantly lower risk with empagliflozin of hospitalization for heart failure or cardiovascular death in adult patients with HFpEF. billion EUR, up 5.8
In 2017, the Food & Drug Administration (FDA) approved the first gene therapy for cancer and for inherited diseases, the first multiplex NGS panel for companion diagnostics (CDx), and the first drug targeting a genetic signature though not a disease. Research through WGS and WES has provided novel insights into biological processes.
These data were shared as a late-breaking oral presentation during the virtual 11 th International AIDS Society Conference on HIV Science (IAS 2021) and are a follow-up to the interim analysis that was presented earlier this year at the virtual 2021 HIV Research for Prevention Conference (HIVR4P 2021). KENILWORTH, N.J.–(BUSINESS
Accelerated approval is an expedited regulatory pathway designed to hasten the availability of drugs (including biologics) that treat serious conditions, offer advantages over existing therapies, and address unmet medical needs. In essence, these trials are the final step that turns provisional approval into full approval.
today announced financial results for the second quarter of 2021. 2021 total revenues guidance reaffirmed at $25.8-$26.6 COVID-19 update : Compared to the first quarter of 2021, we have seen gradual recovery from the impacts of the COVID-19 pandemic. As a result, we expect a 50-60% year-over-year Parsabiv sales decline in 2021.
Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that new data from its haemophilia A clinical programme will be presented at the virtual International Society on Thrombosis and Haemostasis (ISTH) 2021 Congress, from 17-21 July 2021. Roche’s Chief Medical Officer and Head of Global Product Development.
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.
Positive high-level results from the FoCus Phase III trial in Wilson disease showed ALXN1840 met the primary endpoint with a statistically significant improvement in daily mean copper mobilisation from tissues, demonstrating superiority compared with standard-of-care (SoC) treatments.
The favourable tolerability of eliapixant in this trial is consistent with earlier clinical findings. Currently, no treatment is approved for the therapy of RCC, and the medical need for new effective treatments is high. “The As a result, eliapixant may have the potential for safe and effective long-term use.
Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced the presentation of data from the company’s HIV clinical development program at the 11th International AIDS Society Conference on HIV Science (IAS 2021) from July 18-21. Week 96 Metabolic and Bone Outcomes of a Phase 2b Trial of Islatravir and Doravirine.
Onchilles Pharma has translated this breakthrough research into a proprietary set of therapeutics and plans to start first-in-human clinical trials for its first programme, N17350, in 2024 in skin cancers, head and neck cancer, and triple-negative breast cancer. N17350’s mechanism of action delivers three unique attributes for a cancer drug.
today announced that data reinforcing the potential of immunotherapies to impact clinical outcomes for patients will be featured at the European Society for Medical Oncology (ESMO) 2021 Virtual Congress from September 16-21. Summary of Presentations. All times noted are Central European Summer Time (CEST). Abstract Title. Presentation.
State-of-the-art cryogenic freezers alongside material transfer equipment have been installed that are designed to retain the integrity of investigational advanced therapy products by minimizing their time-out-of-environment. With sites in the U.S.,
Paris, April 28, 2021. Strong increase in Q1 2021 business EPS ( 1) at CER. Q1 2021 sales increase of 2.4% Q1 2021 business EPS ( 1) growth at CER driven by efficiency and sales performance, supported by a one-time payment. Sanofi has become a member of the top five companies of the 2021 Access to Medicine index.
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