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2019 Therapies Trials 
BioPharma Drive: Drug Pricing
AUGUST 28, 2023
The treatment, developed by a biotech Bayer acquired in 2019, appeared safe in a small trial and showed signs of an effect on disease progression.
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Drug Target Review
NOVEMBER 17, 2023
They do not induce ‘graft versus host’ disease when transplanted for allogeneic therapy and there appears little sign of immune rejection. Peripheral blood NK cells have been used in allogeneic cancer therapies and shown to be safe. CAR-NK cell trials only commenced in 2017 and most are still recruiting patients.
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Broad Institute
FEBRUARY 26, 2025
These gene signatures provide a roadmap that the field can use to study myeloid cells and how they impact the way brain tumors respond to therapy, Bernstein said. Bradley Bernstein , an institute member at the Broad and chair of the cancer biology department at DFCI, was the studys senior author.
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PLOS: DNA Science
OCTOBER 19, 2023
Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.
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Antidote
JULY 29, 2022
billion to bring a new therapy to market. In 2019 alone, research and development spending totaled $186 billion for the global pharmaceutical industry. Between 2009 and 2018, U.S.
Drug Target Review
FEBRUARY 28, 2024
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.
105 
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
MARCH 25, 2021
this week announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) approved Breyanzi (lisocabtagene maraleucel: liso-cel), a CD19-directed chimeric antigen receptor (CAR) T cell therapy for the treatment of patients with relapsed or refractory (R/R) large B-cell lymphoma 1 and R/R follicular lymphoma. Bristol Myers Squibb K.K.
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Advarra
AUGUST 8, 2023
Gene therapy research is booming in the clinical setting. In this blog, we summarize the growth, risks, and regulatory requirements for gene therapy research. Defining the Boom in Gene Therapy Research The gene therapy field is experiencing explosive growth in today’s competitive research environment.
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The Pharma Data
AUGUST 28, 2020
The Phase 1 trial will be conducted by MindMed later this year and will be the first to investigate what happens when you mix the two drugs to work as a therapy. Researchers are set to investigate a combined dose of LSD and MDMA to treat patients undergoing psychotherapy.
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.
Drug Target Review
OCTOBER 26, 2023
Genome engineering and gene therapies that manipulate DNA sequences in cells have driven a biotechnological revolution over the past decade. Endgame: glybera finally recommended for approval as the first gene therapy drug in the European union. Molecular Therapy 20 , 1831-1832 (2012). Russell S, et al. Wang D, Tai PW, Gao G.
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The Pharma Data
AUGUST 4, 2020
Therapy has been approved to treat advanced NTRK fusion-positive solid tumours. The approval is based on results from the pivotal phase 2 STARTRK-2, phase 1 STARTRK-1 and phase 1 ALKA-372-001 trials, as well as data from the phase 2/3 STARTRK-NG study. Source link.
52 
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.
105 
The Pharma Data
SEPTEMBER 12, 2020
Food and Drug Administration to expand the enrollment of their Phase 3 pivotal COVID-19 vaccine trial to up to approximately 44,000 participants which also allows for the enrollment of new populations. The pivotal trial is event-based and there are many variables that will ultimately impact read-out timing.
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The Pharma Data
JANUARY 17, 2021
18, 2021 /PRNewswire/ — Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Lonza announced today their agreement in support of ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD). NEW YORK , Jan.
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The Pharma Data
MAY 31, 2022
the completion of enrollment of its Phase 1 (Ph1), open-label trial of pluripotent stem cell-derived dopaminergic neurons in patients with Parkinson’s disease (PD). Ph1 clinical trial is to evaluate the safety, tolerability, and?preliminary?efficacy?of AG, announced?the The purpose of the?Ph1 preliminary?efficacy?of F.C.A.H.S. (PI)
52 
The Pharma Data
APRIL 3, 2021
The CoVIg-19 Plasma Alliance today announced that the Phase 3 Inpatient Treatment with Anti-Coronavirus Immunoglobulin (ITAC) clinical trial sponsored and funded by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), did not meet its endpoints. About the ITAC Trial.
The Pharma Data
DECEMBER 5, 2020
.–(BUSINESS WIRE) December 5, 2020 — Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, today announced new clinical data from its Phase 2 trial evaluating SY-1425, its first-in-class selective retinoic acid receptor alpha (RAR?)
The Pharma Data
AUGUST 11, 2020
.–( BUSINESS WIRE )– Bristol Myers Squibb (NYSE: BMY) today announced the Phase 3 CheckMate -577 trial evaluating Opdivo (nivolumab) as an adjuvant therapy for patients with resected esophageal or gastroesophageal junction (GEJ) cancer met its primary endpoint of disease-free survival (DFS) at a pre-specified interim analysis.
52 
The Pharma Data
APRIL 19, 2022
Kite’s Global CAR T-Cell Therapy Manufacturing Network Increasing Capacity by 50% to Meet Patient Demand for New Cancer Therapies. — Scalable and Adaptable Facility Provides Flexibility for Current and Future Cell Therapy Innovation. The site will produce Kite’s FDA approved CAR T-cell therapy used to treat blood cancer.
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The Pharma Data
NOVEMBER 12, 2020
12 November 2020 — The CALAVI Phase II trials for Calquence (acalabrutinib) in patients hospitalised with respiratory symptoms of COVID-19 did not meet the primary efficacy endpoint. No new safety signal for Calquence was observed in the trials. No new safety signal for Calquence was observed in the trials.
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Drug Target Review
MARCH 3, 2025
In this interview, Professor Schwamborn discusses his research, the promise of brain organoids, and his vision for the future of stem cell-based therapies. ” By using personalised models, researchers can identify subgroups of patients with similar molecular characteristics, potentially leading to the development of targeted therapies.
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Eye on FDA
JULY 5, 2022
Most notably in 2012 the Food and Drug Administration Safety and Innovation Act (FDASIA) was signed into law, bring to bear among other things, breakthrough therapy designation. The most obvious impact is that of COVID-19 to recruiting and maintaining clinical trials. Since that time, there has been a healthy output of new treatments.
The Pharma Data
SEPTEMBER 8, 2020
Phase III clinical trial programme initiated for investigational medicine fenebrutinib, designed to be a highly selective and reversible Bruton’s tyrosine kinase (BTK) inhibitor, in relapsing multiple sclerosis (RMS) and primary progressive MS (PPMS).
The Pharma Data
OCTOBER 21, 2020
In the midst of the global pandemic, the analysis found no antiretroviral therapy interruptions across the entirety of the ongoing clinical development programme for long-acting cabotegravir and rilpivirine. Of those participants who transitioned back to injectables, the median duration of oral therapy was 51 days. Source: GSK .
DrugBank
OCTOBER 4, 2024
The FDA's accelerated approval pathways, such as the Breakthrough Therapy designation and the Priority Review program, have shortened development timelines and reduced costs for certain drugs, making them more attractive acquisition targets. However, such acquisitions also entail significant risks for biotech firms.
The Pharma Data
DECEMBER 2, 2020
Based on the Phase 2 study findings to date, marstacimab may have the potential to offer improved bleed control via subcutaneous injection and potentially eliminate the need for prophylactic factor replacement, providing an enhanced treatment option compared to factor replacement therapy.”. In September 2019, the U.S.
52 
The Pharma Data
DECEMBER 29, 2020
As in earlier outpatient trial, immune status when patients entered the trial was a strong predictor of viral load and clinical outcomes. First antibody therapy to demonstrate anti-viral effect in patients hospitalized with COVID-19. Regeneron Pharmaceuticals, Inc. futility analysis). Yancopoulos , M.D.,
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The Pharma Data
DECEMBER 1, 2020
NASDAQ: AXSM), a biopharmaceutical company developing novel therapies for the management of central nervous system (CNS) disorders, today announced positive results from the open-label Phase 2 COMET-TRD trial of AXS-05 in patients with treatment resistant depression (TRD). AXS-05 was well tolerated in the COMET trial.
52 
The Pharma Data
NOVEMBER 20, 2020
NASDAQ: REGN) today announced that the antibody cocktail casirivimab and imdevimab administered together (formerly known as REGN-COV2 or REGEN-COV2), a therapy currently being investigated for use in COVID-19 , has received Emergency Use Authorization (EUA) from the U.S. TARRYTOWN, N.Y., November 21, 2020 – Regeneron Pharmaceuticals, Inc.
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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. SVP, Chief Medical Officer, Novartis Gene Therapies. “We Novartis today announced that the U.S.
The Pharma Data
JANUARY 21, 2021
For one, the patients were not part of a clinical trial that directly tested stem cell transplants against standard MS medications. They all underwent transplants at various Italian medical centers between 1997 and 2019. At the same time, there are important caveats, said Bebo, who was not involved in the study.
52 
Drug Target Review
MAY 18, 2023
Tedesco Distinguished Chair of Paediatric Haematology and Oncology, are collaborating as co-principal investigators on a new clinical trial, which has been awarded a $2.3 The trial represents an early-stage effort to determine the viability of this approach. Kutlar says the possibilities of this additional therapy are exciting.
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Codon
JANUARY 21, 2024
Human challenge trials were an indispensable part of the development of the malaria vaccine, R21/Matrix-M, endorsed by the World Health Organization last October. But for all of their benefits, human challenge trials have their drawbacks. Jake himself has participated in both Zika and Shigella challenge trials.
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The Pharma Data
NOVEMBER 2, 2020
(NASDAQ:ADIL; ADILW) , a clinical-stage biopharmaceutical company focused on the development of treatments for addictions, today provided an update on its landmark ONWARD Phase 3 pivotal trial. 40% of expected Trial patient screenings have been completed and the Trial is more than 25% enrolled relative to planned enrollment.
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The Pharma Data
JANUARY 17, 2021
The clinical trial to evaluate the safety and efficacy of the plasma-derived medicine will have doctors Oriol Mitjà and Bonaventura Clotet as the principal researchers and is expected to begin in early 2021. In 2019, Grifols’ economic impact in its core countries of operation was 8.5 BARCELONA, Spain , Jan.
The Pharma Data
AUGUST 9, 2021
Positive high-level results from the head-to-head DESTINY-Breast03 Phase III trial showed that Enhertu (trastuzumab deruxtecan), the AstraZeneca and Daiichi Sankyo Company, Limited (Daiichi Sankyo) HER2-directed antibody drug conjugate (ADC), demonstrated superiority over trastuzumab emtansine (T-DM1).
52 
Drug Target Review
JUNE 21, 2023
Enabling the systematic discovery of these largely uncharted targets can be a valuable opportunity for development of novel therapies. Genetic-based therapeutics hold significant promise in the clinic Despite the growing number of genes linked to human disease, the proportion of genes targeted by approved therapies remains low.
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The Pharma Data
JANUARY 31, 2021
Clover plans to initiate a global Phase 2/3 efficacy trial of its protein-based S-Trimer COVID-19 vaccine candidate adjuvanted with Dynavax’s CpG 1018 plus alum in the first half of 2021 with an interim analysis for vaccine efficacy potentially in the middle of 2021. EMERYVILLE, Calif. and CHENGDU, China , Feb.
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The Pharma Data
JANUARY 17, 2021
Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases. Launched in 2019, the company raised $700 million in June 2020 in its initial financing. Of course, this is how biotech investments work.
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The Pharma Data
SEPTEMBER 2, 2020
NICE concluded that clinical trial evidence shows that, for people with untreated advanced RCC, Bavencio plus axitinib increases how long people live without their disease getting worse compared with sunitinib, while early data also suggest the therapy increases survival. Source link.
Drug Target Review
AUGUST 24, 2023
Neutralising GDF-15 with CatalYm’s anti-GDF-15 antibody visugromab was shown to reverse its inhibitory effects and to re-sensitise tumours to anti-PD-1 treatment, achieving commensurate survival benefit of anti-GDF-15-anti-PD-1 combination therapy in vivo. 2019 May 22;10:1078. in North America) in late 2017. Frontiers in Immunology.
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The Pharma Data
AUGUST 25, 2020
IDHIFA is the first and only FDA-approved therapy for patients with R/R AML and positive for an IDH2 mutation, which represents up to 19 percent of AML patients. If differentiation syndrome is suspected, initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution. IDHIFA is also approved in Australia and Canada.
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