The Pharma Data

AUGUST 29, 2020

Inclisiran is currently under review by the FDA and the EMA for the treatment of primary hyperlipidemia (including Heterozygous Familial Hypercholesterolemia) in adults who have elevated LDL-C while being on a maximally tolerated dose of statin therapy. 30% reduction of their LDL-C levels with a mean reduction of 54.1% a statin or ezetimibe).

Treatment 52

Treatment Disease Therapies Trials 52

Drug Target Review

AUGUST 9, 2023

14 TDP-43 is a DNA and RNA binding protein that regulates the expression and splicing of several target transcripts. Many potential toxic effects have been suggested, including the mis-splicing of the Stathmin-2 RNA transcript. ALS Therapy Development Institute, www.als.net/news/frontotemporal-dementia-vs-als/. 31, 2016, pp.

RNA 98

RNA Regulations Disease DNA 98

New Drug Approvals

SEPTEMBER 7, 2024

Coronaviruses are enveloped, positive-sense, single-stranded RNA viruses. The genomic RNA of CoVs has a 5′-cap structure and 3′-poly-A tail and contains at least 6 open reading frames (ORFs). Although compounds have been reported to inhibit 3CLpro activity, they have not been approved as coronavirus therapies. 2012, 55, 9069).

RNA 62

RNA Virus Therapies Pharmaceuticals 62

Codon

JUNE 11, 2024

Their solution was to fuse hairpins, little loops made from RNA or DNA, at various positions along the DNA strand that was being sequenced. In 2012 , they found it in the form of DNA polymerase, an enzyme that replicates the genome prior to cell division. More of his writing can be found at stephenmalina.com and an1lam.substack.com.

DNA 73

DNA Engineering RNA Small Molecule 73

Broad Institute

JANUARY 30, 2024

In 2012, he joined the Broad Institute as a postdoctoral researcher in the lab of Broad core institute member Deborah Hung , who is also a professor at Harvard Medical School and MGH, an infectious disease physician at BWH, and an attending critical care physician in the Medical Intensive Care Unit at BWH.

Hospitals 137

Hospitals Medical Schools Virus Science 137

Codon

MARCH 12, 2023

The central dogma is often depicted as DNA→RNA→protein, but it’s much more: A biophysical marvel inside the smallest of vessels. Related news : “More than 200 people have been treated with experimental CRISPR therapies.” Lots of gene therapy data this week. Biology is a Burrito.

DNA 52

DNA Engineering Therapies RNA 52

Codon

MARCH 24, 2024

Water accounts for 70 percent of a bacterium by mass; the other 30 percent includes everything else: proteins, RNA, DNA, lipids, and so on. And given that RNA and lipid nanoparticle synthesis is highly efficient compared to typical bioproduction, it’s likely that the company could have scaled to billions of doses.

Engineering 85

Engineering Vaccine Production RNA 85

Codon

JUNE 26, 2023

Since the last Codon Digest, I’ve published: Reasons to Be Grateful for Biotechnology (with Avadhoot Jadhav) AAV Foundations (Part I) An overview of AAV-based gene therapies, how they get made, and where they go wrong. Nevertheless, phage therapy did have a stint in the U.S. I’ll post an update soon. coli in mice.

DNA 52

DNA Therapies Engineering Clinical Trials 52

Codon

JUNE 26, 2023

Since the last Codon Digest, I’ve published: Reasons to Be Grateful for Biotechnology (with Avadhoot Jadhav) AAV Foundations (Part I) An overview of AAV-based gene therapies, how they get made, and where they go wrong. Nevertheless, phage therapy did have a stint in the U.S. I’ll post an update soon. coli in mice.

DNA 52

DNA Therapies Engineering Clinical Trials 52

PLOS: DNA Science

DECEMBER 21, 2023

Martin’s Press was about to publish my book about gene therapy in 2012, my agent urged me to start blogging. The book that kickstarted DNA Science was The Forever Fix: Gene Therapy and the Boy Who Saved It. Was I Overly Hopeful About Gene Therapy? FDA has approved only a handful. Results can vary.

DNA 105

DNA Vaccine Science Therapies 105

Codon

AUGUST 10, 2023

Link A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity , by Jinek M. PNAS (2012). Link In vitro integration of ribosomal RNA synthesis, ribosome assembly, and translation , by Jewett M.C. Nature (2012). Nature (2012). & Charpentier E. Science (2018). Nature (2005).

DNA 52

DNA Cell Biology Engineering Molecular Biology 52

Codon

AUGUST 10, 2023

Link A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity , by Jinek M. PNAS (2012). Link CRISPR-Cas →CRISPR-Cas: biology, mechanisms and relevance , by Hille F. & & Charpentier E. Philosophical Transactions of the Royal Society B (2016). Science (2018). Nature Biotechnology (2021).

DNA 91

DNA Cell Biology Molecular Biology Engineering 91

The Pharma Data

OCTOBER 30, 2020

Veklury is a nucleotide analog RNA polymerase inhibitor and works by stopping replication of SARS-CoV-2. A pediatric indication was also updated for patients with refractory cHL, or cHL that has relapsed after two or more lines of therapy. It is widely available in U.S. hospitals. In the U.S.,

FDA Approval 52

FDA Approval Hospitals Treatment FDA 52