2026, Disease and Drug Development - Drug Discovery Digest

Taysha Gene Therapies Receives Rare Pediatric Disease and Orphan Drug Designations for TSHA-105 for the Treatment of Epilepsy Caused by SLC13A5 Deficiency

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

Therapies Disease Treatment FDA

Antibody Drug Conjugates: windows of opportunity

Drug Target Review

JULY 4, 2023

The global ADC market is expected to continue this steep upward trajectory and is anticipated to exceed $16 Billion by 2026. In practice, some patients may be left managing a syndrome associated with the therapy along with the disease. 3D rendering of Antibody Drug Conjugate Molecules. Cancer Cell [Internet].

Small Molecule

Small Molecule Drugs Clinical Pharmacology Trials

Ionis highlights achievements, commercial strategy and technology advancements at Investor Day

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

RNA Pharmaceuticals Production Disease

Analysis Life Sciences Thank You What We Expect the FDA to do in August and September 2024

Agency IQ

AUGUST 2, 2024

We expect the FDA to mark the occasion, especially since drug pricing continues to be such a potent issue in the Presidential election. While this would not affect existing vouchers, it would prevent FDA from issuing new ones after September 2026.

FDA

FDA Science Regulations Biosimilars

Sandoz strengthens pipeline expansion through partnership to develop and manufacture multiple biosimilars

The Pharma Data

MAY 15, 2023

Development of the biosimilars will ramp-up over the coming 12-18 months.” Just – Evotec Biologics has developed an optimized proprietary ecosystem for cost-efficient, state-of-the-art drug development and manufacturing. Work is set to begin this year, with full operations provisionally planned for late 2026.

Article FDA Thank You What’s on FDA’s new Diagnostics Chief’s mind? Transitions, the LDT rule and cybersecurity

Agency IQ

AUGUST 23, 2024

The rule has significant implications for the way that LDTs are regulated in general, but also has additional impact on the use of LDTs in the context of drug development (i.e., There are several high-level takeaways from these updates for drug developers.

FDA

FDA Laboratories Regulations Clinical Trials

Fibrosis research: Advances and challenges

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

Small Molecule Research Pharma Companies Disease

Preparing for new-era drug modalities with technology

Drug Discovery World

OCTOBER 13, 2022

Samsung Biologics’ Senior Vice President, James Park, explores the opportunities emerging for CDMOs to support mRNA developers as they explore new therapeutic areas for patients. . One aspect that makes mRNA technology popular with drug developers is that its proteins can be easily edited. billion by 2026 9.

Vaccine

Vaccine Therapies Drugs RNA

Synthetic biology: Shaping the future of drug discovery

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. BCC Research reports that the global market for synthetic biology is estimated to increase from $9.5

DNA

DNA Drugs Engineering Production

Scaling up: The need for human cells to give better access to cell therapies

Drug Discovery World

FEBRUARY 26, 2024

Cell therapies have emerged as transformative and potentially curative treatments across a range of disease areas. Perhaps the greatest example of this has come from CAR-T cell therapies, which have developed as treatments for aggressive lymphomas, particularly in childhood cancers.

Therapies

Therapies Treatment Engineering Disease

Models of Life

Codon

SEPTEMBER 29, 2024

The earliest ones relied on simple linear regression and attempted to correlate genetic variations with observable traits or disease risks — such as drug metabolization rates or cancer susceptibility. It wasn’t a silver bullet to the hard problem of drug development, but it wasn’t too far off either.

Engineering

Engineering Virus DNA Therapies

Analysis Life Sciences Thank You 166 guidance documents the FDA is actively working on in 2024 (and beyond)

Agency IQ

FEBRUARY 15, 2024

If you’re looking for something specific, we recommend using the search function in your browser (Ctrl-F) to look for keywords of interest.

FDA

FDA Science Biosimilars Production

Article EMA Thank You What will the orphan drug market exclusivity haircut mean for industry?

Agency IQ

JUNE 9, 2023

The problems: Although the current regulation stimulated orphan drug research, it didn’t do enough The regulation fostered development of medicines for rare diseases since implementation, but not enough. However, 95% of the over 6,000 recognized orphan diseases still have no treatment options. member states.

Marketing

Marketing Biosimilars Regulations Drugs

Advances in neuroscience drug discovery

Drug Discovery World

FEBRUARY 22, 2024

Dementia is the only major cause of death without a treatment to prevent, slow or stop disease progression. “In With the breadth and range of central nervous system disorders – for example Parkinson’s disease and multiple sclerosis – facing the global population there is an unmet need within this area of drug discovery.

Drugs

Drugs Clinical Trials Disease Therapies

Article FDA Thank You Diagnostics landscape analysis: Key issues and a look ahead at 2024

Agency IQ

FEBRUARY 2, 2024

BY LAURA DIANGELO, MPH | JAN 30, 2024 10:25 AM CST Diagnostic regulation: A quick overview Diagnostics products are those intended to identify, signal or detect a specific health condition, infection or disease, or monitor a person’s health or health status. The diagnostics landscape in the U.S.: In-house tests (similar to LDTs in the U.S.,

FDA

FDA Regulations Laboratories Production

Drug Discovery Digest

Taysha Gene Therapies Receives Rare Pediatric Disease and Orphan Drug Designations for TSHA-105 for the Treatment of Epilepsy Caused by SLC13A5 Deficiency

Antibody Drug Conjugates: windows of opportunity

Trending Sources

Ionis highlights achievements, commercial strategy and technology advancements at Investor Day

Analysis Life Sciences Thank You What We Expect the FDA to do in August and September 2024

Sandoz strengthens pipeline expansion through partnership to develop and manufacture multiple biosimilars

Article FDA Thank You What’s on FDA’s new Diagnostics Chief’s mind? Transitions, the LDT rule and cybersecurity

Fibrosis research: Advances and challenges

Preparing for new-era drug modalities with technology

Synthetic biology: Shaping the future of drug discovery

Scaling up: The need for human cells to give better access to cell therapies

Models of Life

Analysis Life Sciences Thank You 166 guidance documents the FDA is actively working on in 2024 (and beyond)

Article EMA Thank You What will the orphan drug market exclusivity haircut mean for industry?

Advances in neuroscience drug discovery

Article FDA Thank You Diagnostics landscape analysis: Key issues and a look ahead at 2024

Stay Connected