Disease and Trials - Drug Discovery Digest

FDA lifts hold on PTC Huntington’s disease trial

BioPharma Drive: Drug Pricing

JUNE 20, 2024

The agency has lifted a partial trial suspension based on one-year data showing PTC’s pill suppressed a key protein associated with the disorder.

Trials

Trials Disease FDA

How the right technology and tools can accelerate progress in rare disease clinical trials

BioPharma Drive: Drug Pricing

AUGUST 7, 2023

The need for more research into rare diseases is clear, but researchers face significant challenges. Fortunately, technology has created opportunities to innovate in clinical trials for rare diseases.

Clinical Trials

Clinical Trials Disease Trials Research

Xenon gas could protect against Alzheimer's disease: Mouse study

Science Daily: Pharmacology News

JANUARY 15, 2025

Most treatments being pursued today to protect against Alzheimer's disease focus on amyloid plaques and tau tangles that accumulate in the brain, but new research points to a novel -- and noble -- approach: using Xenon gas. A phase 1 clinical trial of the treatment in healthy volunteers will begin in early 2025.

Disease

Disease Clinical Trials Treatment Trials

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Bridging Innovation & Patient Care: The Growing Role of AI

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Novel bone marrow transplant can cure sickle cell disease, study suggests

Science Daily: Pharmacology News

FEBRUARY 25, 2025

A bone marrow transplant process is safe and curative for adults with sickle cell disease, according to results of a trial completed at about 20 cancer centers.

Disease

Disease Trials

Bridging Innovation & Patient Care: The Growing Role of AI

Speaker: Simran Kaur, Co-founder & CEO at Tattva.Health

AI is transforming clinical trials—accelerating drug discovery, optimizing patient recruitment, and improving data analysis. As AI-driven innovation reshapes the clinical trial process, it’s also influencing broader healthcare trends, from personalized medicine to patient outcomes. But its impact goes far beyond research.

Clinical Trials

New Antibiotic, Zoliflodacin, Seeking Approval, Tackles Drug-Resistant Gonorrhea in Trial

Drugs.com

NOVEMBER 3, 2023

3, 2023 -- The first new antibiotic for gonorrhea — the second most common sexually transmitted disease — has shown promise in a clinical trial. FRIDAY, Nov. That news should come as a relief to public health experts, because gonorrhea.

Trials

Trials Clinical Trials Disease Drugs

ALS drug effectively treats Alzheimer's disease in new animal study

Science Daily: Pharmacology News

APRIL 8, 2025

Food and Drug Administration (FDA) for clinical trials for the treatment of amyotrophic lateral sclerosis (ALS) -- improves neuron health in animal models of Alzheimer's disease, according to a new study. Experimental drug NU-9 -- a small molecule compound approved by the U.S.

Small Molecule

Small Molecule Disease Clinical Trials Drugs

Discovery of 'new rules of the immune system' could improve treatment of inflammatory diseases, say scientists

Science Daily: Pharmacology News

JUNE 18, 2024

The discovery could lead to more targeted treatments, with fewer side-effects, for issues from lengthy COVID infections to autoimmune diseases like multiple sclerosis. Clinical trials in humans are now planned.

Treatment

Treatment Immune Response Disease Clinical Trials

Merck antibody reduces RSV-related disease, hospitalizations in trial

BioPharma Drive: Drug Pricing

OCTOBER 18, 2024

Detailed data from a Phase 2b/3 study of Merck’s treatment clesrovimab should support the company’s efforts to win approval by the 2025-2026 season.

Hospitals

Hospitals Disease Trials Treatment

Twice-a-Year Injection Gives Women Full Protection Against HIV, Trial Finds

Drugs.com

JUNE 24, 2024

MONDAY, June 24, 2024 -- Just two injections a year of a new HIV drug protected young women in Africa from infection with the sexually transmitted disease, new trial results show.In announcing the findings, Gilead Sciences Inc. said its HIV.

Trials

Trials Science Disease Drugs

Experimental GLP-1 Med Might Be Breakthrough Against Fatty Liver Disease

Drugs.com

JUNE 10, 2024

MONDAY, June 10, 2024 -- An experimental 'supercharged' form of popular GLP-1 weight-loss meds could help ease fatty liver disease, a new trial suggests. The drug under development, survodutide, helped up to 83% of patients gain real improvements.

Disease

Disease Trials Drugs

New data showcase promise, growing pains of CAR-T in autoimmune disease

BioPharma Drive: Drug Pricing

JUNE 17, 2024

One expert described trial results presented at EULAR last week as “unprecedented.” ” But reports of relapses in some patients drew questions about the therapies’ ultimate potential.

Disease

Disease Therapies Trials

Genome editing used to create disease resistant rice

Science Daily: Pharmacology News

JUNE 15, 2023

Researchers have used the genome-editing tool CRISPR-Cas to create disease resistant rice plants, according to a new study. Small-scale field trials in China show that the newly created rice variety exhibited both high yields and resistance to the fungus that causes a serious disease called rice blast.

Disease

Disease Trials Research

Experimental Drug Could Be Big Advance Against Kidney Disease

Drugs.com

DECEMBER 19, 2023

19, 2023 -- Early results from a trial of a new kidney disease medication show it significantly reduces levels of a urine marker of kidney damage.The experimental drug -- called BI 690517 for now -- cut levels of the liver protein. TUESDAY, Dec.

Disease

Disease Drugs Trials

More Evidence Gene Therapy Might Cure Sickle Cell Disease

Drugs.com

JUNE 12, 2023

MONDAY, June 12, 2023 -- A clinical trial that’s attempting to discover a cure for sickle cell disease has found a new gene therapy to be safe and successful in four patients. Two of the patients were treated at Cleveland Clinic Children’s in.

Therapies

Therapies Disease Clinical Trials Trials

Leveraging AI Solutions for Clinical Trial Efficiencies

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.

Clinical Trials

Clinical Trials Trials Clinical Research Drug Development

One Neurological Factor Keeps Black, Hispanic Patients From Alzheimer's Clinical Trials

Drugs.com

APRIL 19, 2024

FRIDAY, April 19, 2024 -- Black and Hispanic patients with Alzheimer’s disease are greatly underrepresented in clinical trials, even though they’re more likely to get dementia than whites.However, racial discrimination may not be driving this.

Clinical Trials

Clinical Trials Trials Disease

10 clinical trials to watch in the first half of 2024

BioPharma Drive: Drug Pricing

JANUARY 2, 2024

A non-addictive pain pill faces its definitive test, while study results in ALS, a rare heart disease and lung cancer could have far-reaching implications.

Clinical Trials

Clinical Trials Trials Disease

When is a Confirmatory Trial “Underway” or Conducted with “Due Diligence” Enough for Accelerated Approval? FDA Explains Its New Authorities

FDA Law Blog: Drug Discovery

JANUARY 30, 2025

However, as we note in that post, the design, timing of initiation, and timely conduct of confirmatory trials are also important considerations in FDAs determination of whether accelerated approval is appropriate. This blog post focuses on interpreting these new authorities with respect to timely conduct of confirmatory trials.

FDA

FDA Trials Disease Drugs

CAR-T for lupus: the ‘tip of the iceberg’ for cell therapy in autoimmune disease

BioPharma Drive: Drug Pricing

JANUARY 30, 2024

Since a landmark paper in 2022, drugmakers have begun nearly a dozen trials of cell therapies for lupus, with more set to start. Here’s why their efforts are worth watching.

Therapies

Therapies Disease Trials

'Exceptional' results in phase III leukemia trial

Science Daily: Pharmacology News

DECEMBER 10, 2023

Leukaemia trial shows 'exceptional' results. Personalized treatment for the most common form of adult leukemia helps patients survive for longer and stay in remission, a phase III trial has found.

Trials

Trials Treatment Disease Drugs

How AI will reshape pharma by 2025

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.

Clinical Trials

Clinical Trials Trials Pharmaceuticals Drug Development

3 ways decentralized clinical trials could help advance research on rare diseases

BioPharma Drive: Drug Pricing

NOVEMBER 13, 2023

Explore how decentralized clinical trials (DCTs) can enhance rare disease research in three key ways.

Clinical Trials

Clinical Trials Trials Disease Research

Immunovant data show potential for autoimmune disease drug

BioPharma Drive: Drug Pricing

SEPTEMBER 26, 2023

Much anticipated clinical trial data for Immunovant’s experimental FcRn inhibitor could catalyze business development decisions for its parent, Roivant Sciences.

Clinical Trials

Clinical Trials Disease Science Trials

ASH23: CAR-T for autoimmune disease, real-world questions and sickle cell’s social media mentions

BioPharma Drive: Drug Pricing

DECEMBER 9, 2023

Study results disclosed at the American Society of Hematology meeting Saturday suggested potential for cell therapy in lupus and pointed out drug trial limitations.

Drug Trials

Drug Trials Disease Therapies Trials

ASH23: Pharma branding, Editas’ high bar and clinical trial diversity

BioPharma Drive: Drug Pricing

DECEMBER 11, 2023

Editas had the tall task Monday of convincing ASH attendees its gene therapy for sickle cell disease could improve on Casgevy and Lyfgenia.

Clinical Trials

Clinical Trials Trials Therapies Disease

Obesity drug from Boehringer, Zealand succeeds in MASH trial

BioPharma Drive: Drug Pricing

FEBRUARY 26, 2024

Zealand shares rose by more than one-third on the data, which provide further evidence so-called incretin drugs could help treat people with the liver disease.

Trials

Trials Drugs Disease

After surprise trial failure, ALS doctors brace for one less treatment option

BioPharma Drive: Drug Pricing

MARCH 12, 2024

Physicians are preparing for tough conversations with patients on Amylyx Pharmaceuticals’ drug, Relyvrio, while holding out hope it still may help some with the disease.

Doctors

Doctors Treatment Trials Pharmaceuticals

10 clinical trials to watch in the second half of 2023

BioPharma Drive: Drug Pricing

JUNE 27, 2023

A Duchenne gene therapy faces a crucial test, while highly anticipated study results are expected in lung cancer, obesity and heart disease.

Clinical Trials

Clinical Trials Trials Therapies Disease

Wegovy Cuts Heart Risks by 20% in Large Trial

Drugs.com

NOVEMBER 14, 2023

13, 2023 (Healthday News) -- In a finding that could change the landscape of heart disease care, the wildly popular weight-loss drug Wegovy has proved its mettle in protecting the heart after lowering the risk of cardiac problems in. MONDAY, Nov.

Trials

Trials Disease Drugs

Inoculation against diseased fields

Science Daily: Pharmacology News

NOVEMBER 30, 2023

In a large-scale field trial, plant yield increased by up to 40 percent. A research team has now shown that inoculating the soil with mycorrhizal fungi can help maintain or even improve yields without the use of additional fertilizers or pesticides.

Disease

Disease Trials Research

Verve gets FDA green light to run base editing study in US

BioPharma Drive: Drug Pricing

OCTOBER 23, 2023

The trial, which is ongoing in the U.K. since late last year as the FDA sought more details on Verve’s in vivo treatment for heart disease. and New Zealand, has been on hold in the U.S.

FDA

FDA Trials Treatment Disease

Roche autoimmune disease drug disappoints in closely watched trial

BioPharma Drive: Drug Pricing

MARCH 21, 2024

Though the medicine hit its goal in a Phase 3 study in myasthenia gravis, the results fell short of expectations in a boost to rival drug developers like Argenx and AstraZeneca.

Disease

Disease Trials Drugs Drug Development

FDA Approves New Drug to Treat Alzheimer's, Kisunla

Drugs.com

JULY 2, 2024

TUESDAY, July 2, 2024 -- A new drug to treat Alzheimer's disease was approved by the U.S. In clinical trials, donanemab (Kisunla) modestly slowed the pace of thinking declines among patients in the early. Food and Drug Administration on Tuesday.

FDA Approval

FDA Approval Clinical Trials FDA Drugs

Anti-amyloid drug shows signs of preventing Alzheimer's dementia

Science Daily: Pharmacology News

MARCH 19, 2025

An experimental drug appears to reduce the risk of Alzheimer's-related dementia in people destined to develop the disease in their 30s, 40s or 50s, according to the results of a new study.

Clinical Trials

Clinical Trials Drugs Trials Treatment

Ozempic Can Prevent Heart Trouble in Folks With Kidney Issues

Drugs.com

SEPTEMBER 12, 2024

12, 2024 -- Ozempic and Wegovy can prevent heart problems in overweight and obese people, particularly if they also suffer from kidney disease, a new clinical trial shows.The drugs’ active ingredient, semaglutide, reduced heart h. THURSDAY, Sept.

Clinical Trials

Clinical Trials Trials Disease Drugs

Dyne shares jump on trial data for muscle disease drugs

BioPharma Drive: Drug Pricing

MAY 20, 2024

Updated study results for the biotech’s medicines for myotonic dystrophy Type 1 and Duchenne muscular dystrophy appeared to impress analysts and investors on Wall Street.

Disease

Disease Trials Drugs

Cholesterol Med, Fenofibrate, Might Slow Vision Loss in People With Diabetes

Drugs.com

JUNE 25, 2024

TUESDAY, June 25, 2024 -- A well-established cholesterol-lowering drug appears to significantly slow the progression of a diabetes-related eye disease, a new trial shows. Fenofibrate (Tricor) has been approved since 2004 as a means of lowering.

Trials

Trials Disease Drugs

Genetic Matchmaker: Finding Patients for Rare Disease Trials

Fierce BioTech

SEPTEMBER 5, 2024

We will explore how incorporating comprehensive genetic services, like genetic testing access programs and clinical trial matching services, can increase identification of patients who meet program-specific inclusion/exclusion criteria, helping more patients benefit from life-changing therapeutics. Click here to login.

Trials

Trials Disease Clinical Trials Therapies

Another Study Finds CRISPR Gene Therapy Fights Sickle Cell

Drugs.com

JUNE 18, 2024

TUESDAY, June 18, 2024 -- There's more good news in the battle against sickle cell disease, with another trial finding CRISPR gene-editing therapy delivering impressive results for patients.“It’s It’s encouraging that this gene-editing treatment con.

Therapies

Therapies Trials Treatment Disease

FDA Approves New Drug to Treat Alzheimer's

Drugs.com

JULY 2, 2024

TUESDAY, July 2, 2024 -- A new drug to treat Alzheimer's disease was approved by the U.S. clinical trials, donanemab (Kisunla) modestly slowed the pace of thinking declines among patients in the early. Food and Drug Administration on Tuesday.In

FDA Approval

FDA Approval Clinical Trials FDA Drugs

A true liquid biopsy hub – Menarini Silicon Biosystem

BioPharma Drive: Drug Pricing

JUNE 3, 2024

Liquid biopsy has emerged as a revolutionary tool for clinical trials, offering cellular and cell-free methods that provide valuable insights into disease progression and treatment response.

Clinical Trials

Clinical Trials Trials Treatment Disease

Intellia starts late-stage test of CRISPR therapy for rare swelling disease

BioPharma Drive: Drug Pricing

OCTOBER 7, 2024

The study, of Intellia’s treatment for hereditary angioedema, is the second Phase 3 trial the biotech has begun testing its “in vivo” gene editing medicines.

Therapies

Therapies Disease Trials Treatment

The rising impact of biomarkers in early clinical development

Drug Target Review

APRIL 7, 2025

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. A surrogate endpoint is a marker used in clinical trials as a substitute for a direct clinical outcome.

Clinical Development

Clinical Development Clinical Trials Clinical Pharmacology Trials

FDA lifts hold on PTC Huntington’s disease trial

How the right technology and tools can accelerate progress in rare disease clinical trials

Webinars

Trending Sources

Xenon gas could protect against Alzheimer's disease: Mouse study

Webinars

Novel bone marrow transplant can cure sickle cell disease, study suggests

Bridging Innovation & Patient Care: The Growing Role of AI

New Antibiotic, Zoliflodacin, Seeking Approval, Tackles Drug-Resistant Gonorrhea in Trial

ALS drug effectively treats Alzheimer's disease in new animal study

Discovery of 'new rules of the immune system' could improve treatment of inflammatory diseases, say scientists

Merck antibody reduces RSV-related disease, hospitalizations in trial

Twice-a-Year Injection Gives Women Full Protection Against HIV, Trial Finds

Experimental GLP-1 Med Might Be Breakthrough Against Fatty Liver Disease

New data showcase promise, growing pains of CAR-T in autoimmune disease

Genome editing used to create disease resistant rice

Experimental Drug Could Be Big Advance Against Kidney Disease

More Evidence Gene Therapy Might Cure Sickle Cell Disease

Leveraging AI Solutions for Clinical Trial Efficiencies

One Neurological Factor Keeps Black, Hispanic Patients From Alzheimer's Clinical Trials

10 clinical trials to watch in the first half of 2024

When is a Confirmatory Trial “Underway” or Conducted with “Due Diligence” Enough for Accelerated Approval? FDA Explains Its New Authorities

CAR-T for lupus: the ‘tip of the iceberg’ for cell therapy in autoimmune disease

'Exceptional' results in phase III leukemia trial

How AI will reshape pharma by 2025

3 ways decentralized clinical trials could help advance research on rare diseases

Immunovant data show potential for autoimmune disease drug

ASH23: CAR-T for autoimmune disease, real-world questions and sickle cell’s social media mentions

ASH23: Pharma branding, Editas’ high bar and clinical trial diversity

Obesity drug from Boehringer, Zealand succeeds in MASH trial

After surprise trial failure, ALS doctors brace for one less treatment option

10 clinical trials to watch in the second half of 2023

Wegovy Cuts Heart Risks by 20% in Large Trial

Inoculation against diseased fields

Verve gets FDA green light to run base editing study in US

Roche autoimmune disease drug disappoints in closely watched trial

FDA Approves New Drug to Treat Alzheimer's, Kisunla

Anti-amyloid drug shows signs of preventing Alzheimer's dementia

Ozempic Can Prevent Heart Trouble in Folks With Kidney Issues

Dyne shares jump on trial data for muscle disease drugs

Cholesterol Med, Fenofibrate, Might Slow Vision Loss in People With Diabetes

Genetic Matchmaker: Finding Patients for Rare Disease Trials

Another Study Finds CRISPR Gene Therapy Fights Sickle Cell

FDA Approves New Drug to Treat Alzheimer's

A true liquid biopsy hub – Menarini Silicon Biosystem

Intellia starts late-stage test of CRISPR therapy for rare swelling disease

The rising impact of biomarkers in early clinical development

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