The Pharma Data

JANUARY 18, 2021

“There are no approved therapies for epilepsy caused by SLC13A5 that address the underlying cause of this disease,” said RA Session II, President, Founder and CEO of Taysha. “We Taysha’s commitment to developing a potentially life-changing gene therapy for SLC13A5 deficiency is greatly welcomed by our patient community.”.

Therapies 52

Therapies Disease Treatment FDA 52

Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. ADCs have the potential to redress the poor balance between safety and efficacy seen with traditional cancer treatment options. 3D rendering of Antibody Drug Conjugate Molecules.

Small Molecule 145

Small Molecule Drugs Clinical Pharmacology Trials 145

The Pharma Data

DECEMBER 8, 2020

The company’s commercial priorities are three-fold: (1) Initially focusing its commercial efforts on rare diseases within its prolific neurology and cardiology franchises (2) pioneer new markets where there are no available treatments (3) create new standards of care where there has been a lack of innovation to optimize patient care.

RNA 52

RNA Pharmaceuticals Production Disease 52

Drug Discovery World

JANUARY 24, 2024

PW: The big challenge with fibrotic diseases is that they evolve slowly and are diagnosed late in the disease process. This has hindered development of drugs so that, from a therapeutic intervention point of view, fibrosis research is still in its infancy.

Small Molecule 147

Small Molecule Research Pharma Companies Disease 147

Drug Discovery World

FEBRUARY 26, 2024

Reece Armstrong speaks to Kathryn Golden , SVP Technical Operations and Manufacturing, bit.bio, about novel technologies, and methods to manufacture human cells for therapies so more patients can access these treatments. Cell therapies have emerged as transformative and potentially curative treatments across a range of disease areas.

Therapies 130

Therapies Treatment Engineering Disease 130

Drug Discovery World

NOVEMBER 14, 2022

billion by 2026, at a compound annual growth rate (CAGR) of 28.4% during the forecast period of 2021-2026. Just taking a small snapshot of the current sector shines a light on the depth of development taking place. With reports of the drug being prescribed to President Biden, the drug recently hit the headlines. .

DNA 130

DNA Drugs Engineering Production 130

Agency IQ

JUNE 9, 2023

Commission Regulation (EC) No 847/2000 set the rules, defining an orphan drug as one “that it is intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting not more than five in 10 thousand persons in the Community when the application is made.”

Marketing 40

Marketing Biosimilars Regulations Drugs 40