The Pharma Data

JUNE 11, 2025

This mechanism has the potential to partially restore UBE3A protein production in neurons, addressing the root cause of the disorder. Encouraging results from the previously completed Phase 1/2 HALOS study support the continued development of ION582.

Trials 40

Trials Therapies Treatment Disease 40

The Pharma Data

OCTOBER 14, 2020

Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases.

Pharmaceuticals 52

Pharmaceuticals RNA Small Molecule Protein Production 52

Agency IQ

APRIL 12, 2024

Cell-based assays , while more complex to develop and use, offer another option for characterizing product activity at a slightly higher level, allowing sponsors to measure the further downstream effects of products (e.g., morphological cell changes or alterations in protein production or expression).

Production 40

Production FDA Therapies Biochemical Assays 40

Codon

MARCH 20, 2023

Peroxisomal metabolic coupling improves fatty alcohol production from sole methanol in yeast. Optogenetic closed-loop feedback control of the unfolded protein response optimizes protein production. Metabolic Engineering. Assembly of metabolons in yeast using Cas6-mediated RNA scaffolding. ACS Synthetic Biology.

Virus 52

Virus RNA Engineering Small Molecule 52

Codon

MARCH 24, 2024

Once approved, patent-protected drugs are the highest-margin physical products on the planet—even with the high price of bioreactors. The real cost is clinical development. In 2012, an Israeli company called Protalix gained approval for their plant-based enzyme product for Gaucher’s disease. Why rock the boat?

Engineering 87

Engineering Vaccine Production RNA 87

The Pharma Data

JANUARY 24, 2021

Elasmogen is unique in its capacity to isolate and develop soloMERs, with a multi-layered IP position covering the platform, humanisation, formats, products and process including over 25 patents granted in the US, Europe and other territories.

Research 40

Research Virus Medical Schools Government 40