From Fantasy To Reality: Xenotransplantation

Emily BurkeCocktail Fodder, Deoxyribonucleic Acid (DNA), Genomics

TRANSPLANTING ORGANS FROM ANIMALS INTO HUMANS Every ten minutes, a new person is added to the national transplant waiting list. A little more than 75,000 people are active waiting list candidates — meaning they are medically eligible for transplantation according to the Organ Procurement and Transplantation Network. Over the past decade, the gap between organ supply and demand has continued to grow; … Read More

The Science Of CRISPR/Cas9

Emily BurkeBiologics, Business of Biotech, Cancer, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Genetics, Genomics, Mechanism of Action, Ribonucleic Acid (RNA)

CRISPR/CAS9 UPDATE As CRISPR/Cas9 adds new indications to its resume, legal battles over its IP continue to be waged in the US and Europe. On the clinical front, CRISPR/Cas9 entered its first human trial at Sichuan University (Chengdu, China) last fall for metastatic lung cancer, and is widely expected to do so in the U.S. by the end of the year. This … Read More

Epigenome: Writing, Reading & Erasing

Emily BurkeCancer, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Genomics, Mechanism of Action, Small Molecule Drugs

FOUNDATIONS OF EPIGENETICS Genetic mutations — changes in the order of the A, C, G, and T nucleotide bases that make up a gene — have been the primary focus of cancer researchers over the last several decades. By sussing out mutations involved in regulating cell growth and division, scientists better understand the molecular range of different cancers and consequently develop … Read More

Decoding Your Genes

Emily BurkeBiotech Basics, Deoxyribonucleic Acid (DNA), Diagnostics, Drug Approvals, Easily Confused, FDA, Genetics, Genomics, Orphan Disease, Term of the Week, The WEEKLY

The Skinny On DNA Testing 23andMe (Mountain View, CA) recently found itself back in the limelight after the disease risk section of its mail-in DNA kit received an OK from the FDA. The Silicon Valley biotech had to halt sales of its direct-to-consumer genetics testing back in 2013 after regulatory officials grew concerned over marketing claims and the possibility of consumers misinterpreting the test results. 23andMe rebooted a limited part of … Read More

The Science Of CRISPR/CAS9

Emily BurkeBiologics, Business of Biotech, Cancer, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Genomics, Mechanism of Action, Ribonucleic Acid (RNA), The WEEKLY

 CRISPR/CAS9 UPDATE CRISPR/Cas9 can’t seem to stay out of the news — from first in human to patent disputes, we here at the WEEKLY want to update you on this hot technology. A group of scientists from the State Key Laboratory of Proteomics (Beijing, China) and the National Center for Protein Sciences (Beijing, China) recently reported the first ever edit using CRISPR/Cas9 in healthy human … Read More

Stem Cell Snapshot

Emily BurkeBiologics, Cardiovascular Disease, Clinical Trials, Drug Development, Drug Targets, Genetics, Genomics, Mechanism of Action

Inducing Stem Cells To Heal Headlines touting stem cells often claim the therapies heal everything from hair loss to hearing loss. While many of these treatments are not FDA approved, there are some promising innovations winding through preclinical and clinical development. Here at WEEKLY headquarters, we like to tease out the science behind the scene, so let’s review regenerative medicine basics and survey … Read More

New Hope For Spinal Muscular Atrophy

Emily BurkeAntisense, Biologics, Clinical Trials, Drug Development, Drug Targets, FDA, Genomics, Mechanism of Action, Orphan Disease, Orphan Drugs, Ribonucleic Acid (RNA), The WEEKLY

FIRST THERAPY APPROVED FOR SMA Squeaking by on December 23rd as the last new drug approval of 2016, Biogen’s (Cambridge, MA) Spinraza now provides hope for the thousands of families affected by a debilitating neuromuscular disorder known as spinal muscular atrophy (SMA). SMA robs people of their ability to walk, eat, and ultimately, breathe. In addition to Spinraza, there are 13 … Read More

Cancer Vaccines & Game Changers

Emily BurkeBiologics, Cancer, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Genomics, Mechanism of Action, Term of the Week

The Elusive Cancer Vaccine The promise of cancer vaccines have proven to be elusive. A new crop of biotechs are hoping to change that by taking advantage of the latest advances in genomics. Scientists are working overtime trying to develop cancer vaccines that train the immune system to recognize and fight an established tumor. In this WEEKLY, we’ll break down the science … Read More

The Race To Beat SMA

Emily BurkeAntisense, Biologics, Clinical Trials, Drug Development, Drug Targets, FDA, Genomics, Mechanism of Action, Orphan Disease, Orphan Drugs, Ribonucleic Acid (RNA), The WEEKLY

Zeroing In On The SMA Pipeline A decade ago, there was only one drug in development for a debilitating neuromuscular disorder known as spinal muscular atrophy (SMA) — robbing people of their ability to walk, eat, and ultimately, breathe. Today, there are 14 therapies making their way through the clinic according to the patient advocacy group Cure SMA. The increase is … Read More

Next-Generation CAR-T

Emily BurkeBiologics, Business of Biotech, Cancer, Clinical Trials, Drug Development, Drug Targets, Genomics, Mechanism of Action, Monoclonal Antibodies, The WEEKLY

The Race Is On Our last WEEKLY—Hacking the Immune Response—unveiled the science behind CAR-T and TCR, two immunotherapies under the microscope of the mainstream press. The well-deserved media attention highlights the ability of these “living drugs” to recognize and obliterate cancers. With all of the early phase clinical success, a few challenges have popped up: • Safety: CAR-T and TCR can … Read More

On The Hunt

Emily BurkeBiotech Basics, Drug Development, Drug Targets, Genetics, Genomics, Mechanism of Action, Orphan Disease, Orphan Drugs, Term of the Week, The WEEKLY

The Hurdles Of Huntington’s The nervous system is an incredibly complex piece of human machinery, stretching to the far reaches of the body while controlling and receiving the nuances of life from a central command station. Just like any part of the human body, the central nervous system (CNS) is affected by various diseases that are sometimes not entirely understood. After … Read More

The Emerging Proteomics Market

Emily BurkeBusiness of Biotech, Cancer, Deoxyribonucleic Acid (DNA), Diabetes, Diagnostics, Drug Targets, Genomics, Term of the Week, The WEEKLY

Picking Apart The Protein Genomics continues to be all the rage in biotech circles—with special kudos to Illumina’s (San Diego, CA) recent ability to obtain an entire human genome sequence in 24-hours for a mere thousand dollars. Our overall understanding of human DNA combined with our ability to determine individual genomes leads to better disease insight, more powerful diagnostics, and a … Read More

Interrupting HIV

Emily BurkeBiologics, Clinical Trials, Drug Development, Drug Targets, Genomics, HIV, Mechanism of Action, Monoclonal Antibodies, Ribonucleic Acid (RNA), Small Molecule Drugs, The WEEKLY

Therapies In HIV Pipeline Medicine has made gigantic strides in understanding and treating the human immunodeficiency virus (HIV) since its emergence in the early 1980s. Thanks to robust drug discovery efforts, today HIV is managed as a chronic disease instead of the death sentence of years past. The secret to HIV’s infectious success lies in its high mutation rate. Developing drug resistance over … Read More

Presenting The New Class Of Transcriptional Therapeutics

Emily BurkeBiotech Basics, Cancer, Deoxyribonucleic Acid (DNA), Drug Development, Easily Confused, Genetics, Genomics, Mechanism of Action, Ribonucleic Acid (RNA), Small Molecule Drugs, Term of the Week, The WEEKLY

DNA: Coding Vs. NON-Coding The completion of the Human Genome Project in 2003 revealed a big surprise: up to 98% of the DNA making up the human genome does not code for proteins! The notion that parts of the genome were non-coding had been circulating for several decades, but when the actual percentage was confirmed it blew the industry’s mind. This … Read More

A Drop Of Biotech

Emily BurkeCancer, Clinical Trials, Cocktail Fodder, Diagnostics, Drug Development, Genomics, HIV, Orphan Disease, Orphan Drugs, The WEEKLY

Basics of Blood Disorders Blood is the carrier of a multitude of fundamental body processes—responsible for delivering vital nutrients/oxygen and for removing wastes. Like the highway exchange feeding a city of life, blood is simply essential. The branch of medicine concerned with the study, diagnosis, treatment, and prevention of blood related diseases is known as hematology. This single therapeutic area covers a broad range … Read More

The Realm Of Next Generation Sequencing

Emily BurkeBiotech Basics, Cancer, Deoxyribonucleic Acid (DNA), Diagnostics, Genomics, The WEEKLY

Breaking Down The Genome A billion dollar budget, a decade of work, and multinational collaboration brought the first human genome sequence—via the Human Genome Project—to the world at the turn of the century. Today, using so-called “next-generation sequencing (NGS),” an individual human genome sequence costs a mere $1,000 and takes 24 hours. Even greater advances in cost saving and time are expected in … Read More