Science Daily: Pharmacology News

JULY 17, 2024

A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those muscles.

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BioPharma Drive: Drug Pricing

JULY 15, 2024

The company claims the results support exploring the possibility of an accelerated approval, citing flexibility by FDA officials in reviewing rare disease gene therapies.

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Drugs.com

JULY 16, 2024

TUESDAY, July 16, 2024 -- Hormone therapy for breast cancer might reduce a woman’s later risk of dementia and Alzheimer’s disease, a new study finds.Overall, hormone therapy is associated with a 7% lower risk of developing Alzheimer’s or a related d.

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Drugs.com

DECEMBER 8, 2023

Food and Drug Administration on Friday approved two milestone gene therapies for sickle cell disease, including the first treatment ever approved that uses gene-editing technology. FRIDAY, Dec. 8, 2023 -- The U.S. Casgevy, developed by Vertex.

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Drug Target Review

JUNE 9, 2025

The landscape of genetic medicine is undergoing a profound transformation, driven by innovative approaches that challenge the traditional, disease-specific paradigms. My transition to biotech and rare diseases was deeply personal – my son was diagnosed with Duchenne muscular dystrophy in 2020. “My

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BioPharma Drive: Drug Pricing

JUNE 8, 2023

Two-year-old Kate Therapeutics is launching publicly with $51 million in funding and a licensing deal with Astellas Pharma for a neuromuscular disease gene therapy.

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BioPharma Drive: Drug Pricing

OCTOBER 10, 2023

The pharma plans for the new Berkeley facility to produce clinical, and potentially commercial, supplies of a Parkinson’s disease cell therapy being tested by its subsidiary BlueRock.

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Science Daily: Pharmacology News

MAY 16, 2024

In an important step toward more effective gene therapies for brain diseases, researchers have engineered a gene-delivery vehicle that uses a human protein to efficiently cross the blood-brain barrier and deliver a disease-relevant gene to the brain in mice expressing the human protein.

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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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Science Daily: Pharmacology News

AUGUST 9, 2023

Promising preclinical results show hematopoietic stem cell therapy was effective in rescuing memory loss, neuroinflammation and beta amyloid build-up in a mouse model of Alzheimer's disease.

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BioPharma Drive: Drug Pricing

JUNE 15, 2023

Lilly will pay the genetic medicine specialist $60 million upfront to work together on a treatment targeting a risk factor known as Lp(a).

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BioPharma Drive: Drug Pricing

JULY 1, 2024

Like many of its cell therapy peers, Artiva, which originally sought an IPO in 2021, has shifted its strategy in hopes of riding a recent wave of investor interest in autoimmune disease research.

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BioPharma Drive: Drug Pricing

SEPTEMBER 18, 2023

Three years after gaining European approval, Libmeldy is now under U.S. review with a deadline set for March.

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BioPharma Drive: Drug Pricing

DECEMBER 17, 2024

Phase 2 results in inflammatory bowel disease hinted the therapy could be more potent than similar medicines from Merck and Roche, each of which were acquired in multibillion-dollar deals.

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Science Daily: Pharmacology News

AUGUST 30, 2023

Scientists have made a `paradigm shifting' discovery on the mechanisms required for learning and memory that could lead to new therapies for Alzheimer's disease and potentially Down syndrome.

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BioPharma Drive: Drug Pricing

MAY 27, 2025

Elsewhere, Angelini Pharma licensed a rare disease therapy and the FDA refused to review a Savara lung drug. City will receive $46 million from Biogen under the alliance.

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BioPharma Drive: Drug Pricing

JUNE 17, 2024

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

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Drugs.com

AUGUST 31, 2023

31, 2023 -- A type of gene therapy that precisely "edits" a key bit of DNA might offer a new way to treat sickle cell disease -- a painful inherited condition that largely strikes Black children and adults. THURSDAY, Aug. That's according to a new.

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Drugs.com

DECEMBER 29, 2023

29, 2023 -- A new cellular therapy improved learning and memory in mice with Alzheimer's disease, researchers report.The therapy -- developed at the University of Nebraska Medical Center (UNMC) -- relies on both the immune system to. FRIDAY, Dec.

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Drugs.com

DECEMBER 8, 2023

Food and Drug Administration on Friday approved two milestone gene therapies for sickle cell disease, including the first treatment ever approved that uses gene-editing technology.Casgevy, developed by Vertex. FRIDAY, Dec. 8, 2023 -- The U.S.

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BioPharma Drive: Drug Pricing

MARCH 10, 2025

While initial study results suggest Beam's technology can correct alpha-1 antitrypsin deficiency's genetic roots, shares fell by double digits.

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Science Daily: Pharmacology News

APRIL 9, 2025

By inserting an entire gene, the tool offers a one-and-done approach that overcomes hurdles from CRISPR gene editing technology -- which is programmed to correct individual mutations -- offering a promising step forward for gene therapy.

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BioPharma Drive: Drug Pricing

MARCH 19, 2024

clearance of Lenmeldy, for a rare and inherited metabolic disease, triggers an additional payout related to Kyowa Kirin’s recent deal to acquire the once high-flying gene therapy developer.

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Science Daily: Pharmacology News

FEBRUARY 14, 2025

One day, researchers want to use switches of this kind to trigger cell therapies for various metabolic diseases. Researchers have developed a new gene switch that can be activated using a commercially available nitroglycerine patch applied to the skin.

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BioPharma Drive: Drug Pricing

JULY 2, 2024

The company claimed Phase 2 study results prove the therapy’s promise treating myasthenia gravis. But shares lost more than a quarter of their value amid questions about the data.

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Chemical Biology and Drug Design

NOVEMBER 4, 2024

Alzheimer's disease (AD) is a progressive age-related neurodegenerative brain disorder that destroys memory and other important mental functions. ABSTRACT Alzheimer's disease (AD) is a progressive chronic age-related neurodegenerative brain disorder characterized by the loss of memory and other cognitive functions.

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BioPharma Drive: Drug Pricing

OCTOBER 10, 2024

Seven young boys given Bluebird's Skysona later developed blood cancers, findings that could shape how doctors balance the gene therapy’s risks against its benefit.

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Science Daily: Pharmacology News

NOVEMBER 13, 2023

This discovery opens up new opportunities for therapeutic intervention to control cholesterol uptake that could complement other therapies and potentially save lives. Researchers have discovered the mechanism by which cholesterol in our diet is absorbed into our cells.

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Broad Institute

MAY 21, 2025

Scientists create next generation of tools in battle against brain disease By Corie Lok May 21, 2025 Breadcrumb Home Scientists create next generation of tools in battle against brain disease The findings contained in eight studies could lead to targeted gene therapies for brain disorders.

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BioPharma Drive: Drug Pricing

FEBRUARY 21, 2024

The full results spotlight cell therapy’s potential to treat lupus and other inflammatory conditions, but also the problems drug developers must solve first.

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Drugs.com

NOVEMBER 28, 2023

28, 2023 -- Stem cells injected into the brains of multiple sclerosis patients appear to protect them against further damage from the degenerative disease, a new study shows.MS TUESDAY, Nov. occurs when the body’s own immune system attacks and.

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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.

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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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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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BioPharma Drive: Drug Pricing

SEPTEMBER 7, 2023

Fresh off a Series C fundraise, Kriya has acquired the privately held Tramotane Therapeutics and its preclinical candidate for the widely prevalent liver disease.

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Chemical Biology and Drug Design

DECEMBER 15, 2023

The figure depicts the neuroprotective role of exendin-4 in Alzheimer's disease and Parkinson's disease. Due to the complex pathological processes underlying neurodegeneration, at present, there is no viable therapy available for neurodegenerative disorders.

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Science Daily: Pharmacology News

JANUARY 3, 2025

The research could revolutionize therapies for complex conditions like autoimmune disease and cancer. Bioengineers have developed a new construction kit for building custom sense-and-respond circuits in human cells.

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