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.

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Drugs.com

AUGUST 14, 2023

14, 2023 -- For people with severe alcohol use disorder, a new gene therapy trial could lead to an effective treatment that would involve chemically rebalancing the area of the brain associated with addiction. MONDAY, Aug. “With alcohol.

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Drugs.com

MAY 6, 2024

MONDAY, May 6, 2024 -- An injectable gene therapy caused measurable improvements in vision among a small group of people with inherited blindness, an early-stage clinical trial says.Researchers recruited 14 people with Leber Congenital Amaurosis.

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Therapies Clinical Trials Trials 290

Science Daily: Pharmacology News

SEPTEMBER 25, 2024

Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71 percent after a single infusion of gene therapy, according to the new results of an international Phase III clinical trial.

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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.

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Drugs.com

JUNE 25, 2024

TUESDAY, June 25, 2024 -- An experimental stem cell therapy can essentially cure type 1 diabetes by restoring insulin production in some patients, early clinical trial results show.Seven out of 12 patients no longer needed daily insulin shots after.

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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.

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BioPharma Drive: Drug Pricing

OCTOBER 4, 2024

An experimental Alzheimer’s therapy from Cassava Sciences is still being tested in two Phase 3 studies, even as the company has come under regulatory scrutiny.

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Trials Science Therapies 360

Science Daily: Pharmacology News

MAY 8, 2024

The study provides both mechanistic and preclinical evidence supporting the rapid initiation of clinical trials for patients with acute myeloid leukemia (AML) to test an existing medicine that inhibits the complex, called eganelisib, both alone and in combination with the most used AML chemotherapy, cytarabine.

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Therapies Clinical Trials Trials 292

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.

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Clinical Trials Trials Clinical Research Drug Development 95

Drugs.com

JUNE 7, 2023

TUESDAY, June 6, 2023 -- Radiation therapy might not be necessary in treating some forms of rectal cancer and lymphoma, sparing patients from the toxic treatment, a pair of new clinical trials shows. One trial found that rectal cancer patients.

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Therapies Clinical Trials Trials Treatment 246

Drugs.com

MAY 29, 2024

WEDNESDAY, May 29, 2024 -- People battling advanced colon cancers might have a new treatment option that could extend their survival, a new trial finds.A combination of two experimental immunotherapy drugs plus standard chemotherapy led to a median.

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Therapies Trials Treatment Drugs 297

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.

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Therapies Virus Trials Hospitals 284

BioPharma Drive: Drug Pricing

JUNE 4, 2024

A group of independent experts wasn't convinced by clinical trial data from company Lykos Therapeutics, which is seeking FDA approval of MDMA-assisted treatment for post-traumatic stress disorder.

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FDA FDA Approval Clinical Trials Therapies 329

Drugs.com

DECEMBER 15, 2023

15, 2023 -- Patients with advanced heart failure can benefit from stem cell therapy, a large, new clinical trial has found.Injections of stem cells programmed to heal damaged heart tissue wound up improving overall quality of life for. FRIDAY, Dec.

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BioPharma Drive: Drug Pricing

NOVEMBER 5, 2024

The companies’ CAR-T seemed to cause fewer movement disorders in a multiple myeloma trial than J&J's Carvykti has in other tests, although Wall Street analysts were divided on how the two drugs compare.

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Therapies Trials Drugs 301

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. Each child had an inherited deafness caused by mutations in the OTOF gene, called DFNB9.

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Therapies Clinical Trials Trials Research 214

BioPharma Drive: Drug Pricing

MAY 23, 2024

The findings, from two trials involving tens of thousands of women, could influence guidelines around the use of hormonal treatment for menopausal symptoms.

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Therapies Trials Treatment 293

Science Daily: Pharmacology News

AUGUST 30, 2023

A clinical trial has tested a potentially curative stem cell gene therapy for sickle cell disease. The results were promising.

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Therapies Clinical Trials Trials Disease 209

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.

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Therapies Treatment Engineering Clinical Trials 65

Science Daily: Pharmacology News

AUGUST 18, 2023

Novel stem cell therapy called a CALEC transplant was performed safely in four patients with serious chemical burns tracked in a phase I clinical trial for 12 months. CALEC procedure takes small amount of stem cells from healthy eye, grows and expands stem cells on a graft, before it is transplanted into the patient's damaged eye.

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BioPharma Drive: Drug Pricing

SEPTEMBER 27, 2023

Expert advisers to the agency voted 17-1 that Brainstorm's clinical trial data did not show the company's stem cell treatment was effective for treating ALS.

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Therapies Clinical Trials FDA Trials 284

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.

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Clinical Trials Trials Therapies Disease 300

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.

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Disease Therapies Trials 349

BioPharma Drive: Drug Pricing

SEPTEMBER 16, 2024

Applying CAR T-cell therapy in new clinical settings presents unique challenges. Worldwide Clinical Trials is at the forefront, partnering with drug development teams to map this new path forward.

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Therapies Trials Clinical Trials Drug Development 130

BioPharma Drive: Drug Pricing

SEPTEMBER 27, 2023

Expert advisers to the agency voted 17-1 that Brainstorm's clinical trial data did not show the company's stem cell treatment was effective for treating ALS.

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Therapies Clinical Trials FDA Trials 274

BioPharma Drive: Drug Pricing

NOVEMBER 9, 2023

Shares in the biotech tumbled by 75% as results from a Phase 2 trial showed more improvement in participants given a placebo.

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Therapies Trials 284

Science Daily: Pharmacology News

FEBRUARY 22, 2024

A large-scale clinical trial of treatment strategies for Crohn's disease has shown that offering early advanced therapy to all patients straight after diagnosis can drastically improve outcomes, including by reducing the number of people requiring urgent abdominal surgery for treatment of their disease by ten-fold.

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Therapies Clinical Trials Treatment Disease 221

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.

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Clinical Trials Trials Therapies Disease 293

Conversations in Drug Development Trends

NOVEMBER 12, 2024

By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations. Operationalizing these trials requires proactive and flawless management at every stage.

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Drugs.com

SEPTEMBER 26, 2024

25, 2024 -- A new gene therapy approved earlier this year can serve as a sustainable single-dose treatment for people with hemophilia B, newly published clinical trial data shows. THURSDAY, Sept. People with hemophilia B saw their bleeding episodes.

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BioPharma Drive: Drug Pricing

OCTOBER 10, 2024

Data readouts over the next six months could set expectations for how the highly lucrative market for weight loss therapies will look in the future.

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Trials Therapies Marketing 273

Science Daily: Pharmacology News

NOVEMBER 27, 2023

An international team has shown that the injection of a type of stem cell into the brains of patients living with progressive multiple sclerosis (MS) is safe, well tolerated and has a long-lasting effect that appears to protect the brain from further damage.

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Drugs.com

DECEMBER 21, 2023

21, 2023 -- A new treatment appears to reduce food allergies in children and teens, according to interim clinical trial results.A THURSDAY, Dec. lab-made monoclonal antibody called omalizumab (Xolair) significantly increased the amounts of common.

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Clinical Trials Therapies Trials Treatment 260

BioPharma Drive: Drug Pricing

SEPTEMBER 11, 2024

Data from an early trial of the therapy fell short of the company’s “threshold to warrant additional capital investment,” an executive said.

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Therapies Trials 176

BioPharma Drive: Drug Pricing

APRIL 16, 2024

Shares of the biotech rose 25% after a large trial found adding its drug Caplyta onto antidepressant therapy significantly helped people with major depression.

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Trials Drugs Therapies 196

BioPharma Drive: Drug Pricing

JUNE 13, 2024

The failure of another trial adds to uncertainty around the benefits of the gene-based treatments, though industry analysts still expect Sarepta’s Elevidys’ to be unaffected.

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Therapies Trials Treatment 244