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Research Therapies Trials 
Science Daily: Pharmacology News
MARCH 4, 2025
Results from a phase 1/2 clinical trial of a novel stem cell treatment for cornea injuries found 14 patients treated and tracked for 18 months had a more than 90% success rate at restoring the cornea's surface and improvements in vision.
296 
Science Daily: Pharmacology News
MARCH 27, 2025
Researchers conducted the first clinical trial of an AI-powered therapy chatbot and found that, on average, people with diagnosed mental disorders experienced clinically significant improvements in their symptoms over eight weeks, according to new results.
291
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Science Daily: Pharmacology News
SEPTEMBER 6, 2024
The vision of people with a rare inherited condition that causes them to lose much of their sight early in childhood was 100 times better after they received gene therapy to address the genetic mutation causing it.
328 
BioPharma Drive: Drug Pricing
MAY 7, 2024
The company is working with trial researchers to investigate further. Pfizer said the patient, a young boy who was treated earlier last year, had died suddenly.
360 
BioPharma Drive: Drug Pricing
APRIL 3, 2025
The Swiss drugmaker, which holds European rights to Elevidys, suspended three trials while researchers investigate the death of a young man who died following treatment.
270 
BioPharma Drive: Drug Pricing
SEPTEMBER 12, 2024
Spun out of research at Nationwide Children’s Hospital, Vironexis claims it will be the first company to start a study testing a cancer drug delivered via the adeno-associated viruses commonly used in genetic medicines.
281 
BioPharma Drive: Drug Pricing
JUNE 22, 2023
The conditional clearance for Sarepta’s Elevidys in 4- and 5-year-olds is a milestone for research into the deadly disease, and raises the stakes of an ongoing trial that could prove how well it works.
327 
PPD
JANUARY 15, 2025
As clinical trials become increasingly complex, particularly in decentralized trials and rare disease studies, sponsors experience increased challenges in site selection, forecasting and resourcing, and patient recruitment and enrollment. Discover how AI is used to optimize key aspects of clinical trial management. Get in touch.
Science Daily: Pharmacology News
JANUARY 24, 2024
A novel gene therapy for hearing loss was administered to six children in China in a clinical trial. The researchers report in a new study, after 26 weeks, five children demonstrated hearing recovery and dramatic improvements in speech perception and the restored ability to conduct normal conversation.
218 
Drug Target Review
FEBRUARY 10, 2025
In the rapidly advancing field of cell therapies, Dr Jason Bock has emerged as a leader, known for his innovative approach to optimising the development process. With over 25 years of experience in therapeutics, Bock has played a pivotal role in shaping the future of cell therapies, particularly through his work at CTMC.
64 
Drug Target Review
DECEMBER 20, 2024
However, recent breakthroughs in AI, such as predictive modelling, clinical trial optimisation, and personalised medicine, have demonstrated its potential. We spoke with Aaron Smith, a mathematician-turned-machine learning scientist and the founder of Unlearn , a company leading the charge in applying AI to optimise clinical trial efficiency.
128 
Science Daily: Pharmacology News
MARCH 7, 2024
Four children have remained free of detectable HIV for more than one year after their antiretroviral therapy (ART) was paused to see if they could achieve HIV remission, according to new research.
321 
Drugs.com
SEPTEMBER 14, 2023
14, 2023 -- A new study is adding to evidence that the party drug "ecstasy" can boost the benefits of talk therapy for people suffering from post-traumatic stress disorder (PTSD). In a clinical trial, researchers found that three. THURSDAY, Sept.
279 
Conversations in Drug Development Trends
OCTOBER 14, 2024
Fortunately, advances in clinical research are providing hope for better treatments and outcomes. With the support of global networks like ours at Worldwide Clinical Trials (Worldwide), autoimmune and bone health research is accelerating, providing new solutions to patients around the world.
52 
PPD
NOVEMBER 11, 2024
As a result, biopharma and biotech companies working to bring their drug pipelines to the market require deep expertise from trusted partners to help deliver critical therapies for their patients. Accelerate your drug development and clinical trial goals and benefit from our 360° CDMO and CRO solutions and expertise. billion in 2035.
Broad Institute
JUNE 27, 2024
There are currently no treatments, but researchers from the Whitehead Institute for Biomedical Research and Broad Institute of MIT and Harvard have developed an approach that could one day be used to turn off the gene encoding this protein throughout the brain to treat or even prevent prion disease.
142 
Broad Institute
NOVEMBER 2, 2023
Bayer and Broad Institute extend cancer therapy research collaboration By Corie Lok November 2, 2023 Breadcrumb Home Bayer and Broad Institute extend cancer therapy research collaboration Long-standing industry-academia collaboration has already resulted in three clinical oncology candidates.
105 
Codon
FEBRUARY 13, 2024
Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Goodsell, RCSB Protein Data Bank and Scripps Research. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Always free.
128 
Drug Target Review
DECEMBER 23, 2024
While these therapies hold great promise for improving cancer treatment outcomes, their development presents significant challenges, especially in achieving the optimal balance between efficacy and safety. To explore these challenges further, we spoke with Cheng Wang, a postdoctoral researcher at the University at Buffalo.
52 
Broad Institute
OCTOBER 29, 2024
Related groups Center for the Development of Therapeutics (CDoT) A team of researchers at Broad Institute of MIT and Harvard, in a long-term project that has included industry collaborators at Bayer and Trueline Therapeutics, has developed a compound called BRD-810 that holds promise as a therapeutic candidate for cancer.
133 
BioPharma Drive: Drug Pricing
AUGUST 12, 2024
Technology emerging from Yale researcher Craig Crews’ labs will be used to target prostate and breast cancer in early clinical trials.
164 
SCIENMAG: Medicine & Health
FEBRUARY 26, 2024
The 7th World Conference on Targeting Phage Therapy 2024, two-day event dedicated to advancing the field of phage research and therapy will be hosted at Corinthia Palace Malta on June 20-21.
89 
Drug Target Review
MARCH 4, 2025
Phenomix Sciences, built on over a decade of clinical research at the Mayo Clinic, is disrupting this outdated approach. The research demonstrated that obesity is not one disease, but many; each of which should be treated with different interventions, says Bagnall. Obesity is rising worldwide, driven by lifestyle, diet, and genetics.
52 
Codon
NOVEMBER 3, 2024
Even after microbiologists discovered the bacterium that causes the illness in 1882, it wasn’t until the 1920s that researchers were able to develop a vaccine for TB. A 1994 review of 14 prospective trials and 12 case-control studies revealed that the BCG vaccine reduced the risk of TB by 50 percent. million people every year.
98 
FDA Law Blog: Drug Discovery
NOVEMBER 10, 2024
Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field. In October, FDA announced seven new clinical trial grants awarded in fiscal year (FY) 2024 – including one for a Phase 3 trial – totaling $17.2
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.
118 
PPD
OCTOBER 17, 2024
Cell and gene therapy (CGT) studies are rapidly gaining momentum in the Asia-Pacific region, fueled by growing patient demand and a thriving ecosystem of innovation. In China, the high incidence of solid tumors is driving an urgent need for advanced therapies, spurring the push for new treatment approaches.
97 
Broad Institute
FEBRUARY 26, 2025
Why brain cancer is often resistant to immunotherapy By Allessandra DiCorato February 26, 2025 Breadcrumb Home Why brain cancer is often resistant to immunotherapy Researchers find four coordinated gene expression programs in immune cells from glioma tumors, including two that could lead to immunotherapy resistance.
132 
FDA Law Blog: Drug Discovery
MARCH 13, 2023
Who better than people living with a condition to inform drug companies, physicians, academics, and the FDA on what it is like to live with their condition, what symptoms most impact their lives, what goes into their decision about whether to participate in a clinical trial, and what kind of treatment effects would be most meaningful to them?
98 
Advarra
NOVEMBER 14, 2024
In an era where clinical trials are increasingly global, it’s more imperative than ever to leverage international expertise. Data and safety monitoring boards (DSMBs), also known as data monitoring committees (DMCs), play a critical role in overseeing a clinical trial’s safety and efficacy.
Advarra
MARCH 6, 2025
Institutional review boards (IRBs) play a crucial role in the ever-evolving field of clinical research. Innovations in fields such as immunotherapy, cancer and chronic diseases owe their existence to clinical trials, which judiciously test and evaluate safety and efficacy. Advarra ensures that clinical trials comply with the U.S.
Broad Institute
OCTOBER 11, 2023
Manguso, who’d recently graduated from college and was conducting research at the University of Copenhagen as a Fulbright scholar, moved back to the Boston area to be with his mother as she underwent treatment. By then, a few cancer immunotherapies had entered clinical trials and only a handful had been approved by the FDA.
137 
Drug Target Review
FEBRUARY 9, 2024
What key findings about stem cell behaviour, differentiation and integration within host tissues impact the development of stem cell therapies? Despite their potential, ADSC therapy faces several challenges in preclinical studies. Is there a certain disease or condition that you believe stem cell therapy holds the most promise for?
105 
Antidote
JUNE 7, 2023
Before any new medical treatment, therapy, or device is approved, it must first be tested through clinical research studies. While some research studies seek participants with illnesses or conditions to be studied in the clinical trial, it is not always necessary to have a specific illness or condition to participate.
98 
PPD
MARCH 11, 2025
While individual rare diseases affect populations that are small in numbers, collectively they impact millions globally, posing significant health and research challenges. Moreover, in some rare disease trials, it is unethical to design a control group of patients with a placebo.
Fierce BioTech
NOVEMBER 21, 2023
Cell and Gene Therapy (CGT) has the potential to transform the life trajectory of children with genetic conditions amendable to gene modification with advances in treatment and possible cures. Pediatric CGT trials have the potential to cure diseases and prolong lives.
91 
Antidote
JULY 27, 2023
Before any new therapy can be put on the market, it must be tested to ensure its safety and effectiveness, and the research for this process is done through clinical trials.
PPD
AUGUST 5, 2024
Food and Drug Administration (FDA)-approved therapies for treating PAH were primarily vasodilators, designed to overcome the imbalance between vasoactive and vasodilator mediators and to restore endothelial cell function. As a result, researchers now often use composite endpoints in lieu of the customary single primary endpoint.
DrugBank
AUGUST 9, 2024
Investment Trends in Pharmaceutical Research and Development Investing in the pharmaceutical sector presents a unique set of challenges and opportunities; let’s examine them in more detail. These new therapies have demonstrated significant LDL cholesterol reductions, but their high cost remains a barrier to widespread adoption.
Antidote
OCTOBER 7, 2022
Different types of clinical studies are designed to investigate potential therapies, medications, and medical devices — and all of these must go through each phase of a clinical trial before they are approved.
98 
PPD
AUGUST 7, 2024
Clinical trials are the backbone of medical research, enabling the development of new treatments and therapies that can improve patient outcomes. However, conducting successful clinical trials requires efficient communication and coordination among various stakeholders.
PPD
JUNE 7, 2023
The number of pediatric patients diagnosed with nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH) is on the rise, yet there are currently no approved therapies to treat NAFLD and NASH in adult or pediatric populations. As therapies for the treatment of NASH in adult patients go this year to the U.S.
98 
Drug Target Review
NOVEMBER 29, 2023
Our primary focus is to design and develop RNA therapies for liver diseases. Our team of chemists then uses this information to develop novel therapies. By doing this, we get a superior understanding of which therapies will and will not work in human biology before a clinical trial.
102 
Antidote
AUGUST 29, 2022
In the world of medical research, experts are constantly looking for new drugs, treatments, and therapies for a variety of conditions — but the process of getting these treatments approved is only possible if they are tested through clinical trials.
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