Remove 2018 Remove FDA Approval Remove Small Molecule Remove Therapies
article thumbnail

A new drug approval for the vanguard of RNA-targeted small molecules

Dark Matter Blog

A few years ago, at Arrakis Therapeutics, we set out to conquer a strange new territory, drugging RNA structures with small molecules. On the strength of those findings, nusinersen was approved by the FDA at the end of 2016 for the treatment of SMA , marketed under the commercial brand Spinraza. We will replace it.”

article thumbnail

Taking a new approach to tackle neurodegenerative diseases

Drug Discovery World

Using its proprietary compound screening platform, Oligomerix discovers and develops differentiated, oral, small molecule inhibitors of tau self-association that are easy to administer and cost-effective. . Global dementia prevalence is expected to increase to 152 million in 2050, a 204% increase compared to 2018.

Disease 130
Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

Three trends in the antibody-drug conjugate (ADC) market 

Drug Discovery World

Antibody-drug conjugates (ADCs) are biopharmaceutical products in which a monoclonal antibody (mAB) is linked to a small molecule drug with a stable linker 1. A Nature publication confirmed that there are currently 12 FDA-approved ADCs on the market, and nine of these secured FDA approval in the past six years 2.

Marketing 245
article thumbnail

Analysis Life Sciences Thank You The U.S. regulatory landscape for probiotics and other microbiome-based therapies

Agency IQ

regulatory landscape for probiotics and other microbiome-based therapies Probiotics, a type of microbiome-based therapy containing live micro-organisms, are broadly available on drug store and grocery store shelves, but the regulatory classifications for products containing the same micro-organism can differ dramatically.

article thumbnail

The evolution of the RNA therapeutics landscape 

Drug Discovery World

While mRNA vaccines and RNA interference therapies have garnered much attention, a new class of RNA therapeutics known as aptamers is gaining momentum, particularly to address acute diseases. The most developed RNAi is small interfering ribonucleic acid (siRNA)-based therapies.

RNA 147
article thumbnail

Lineage Cell Therapeutics Provides End of Year Shareholder Letter

The Pharma Data

NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, today provided a year-end review and an outline of its plans for 2021. CARLSBAD, Calif.–( –( BUSINESS WIRE )– Lineage Cell Therapeutics, Inc.

article thumbnail

FDA accepts Pfizer’s Supplemental New Drug Applications for BRAFTOVI + MEKTOVI

The Pharma Data

Food and Drug Administration (FDA) has accepted for review the Supplemental New Drug Applications (sNDAs) for BRAFTOVI® (encorafenib) + MEKTOVI® (binimetinib) for patients with metastatic non-small cell lung cancer (NSCLC) with a BRAF V600E mutation, as detected by an FDA-approved test. In the U.S.,

FDA 40