What Are Chemicals? | How Drugs Work in the Body | Understanding Homeostasis

We’re all made of chemicals—but what exactly is a chemical? In this video, we break down the basics: chemicals are forms of matter that exist as solids, liquids, or gases. Inside living organisms, these chemicals create complex networks that keep us alive and balanced—a process known as homeostasis.

Drugs are chemicals too! They work by interacting with these biological networks to help restore or adjust how the body functions. Whether you’re a curious learner, a patient, or a future scientist, understanding these fundamentals can empower better decisions about your health and treatment.

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Full Intro to Medical Science Playlist: https://www.youtube.com/playlist?list=PLrDVyc3t26Fy5aQpo3mulackGlUwrIqYL

In This Episode, We Explore

- What exactly is a chemical—and why it matters - The definition of a drug and how it works in the body - How chemical reactions power life - Biochemicals: the molecules that make living systems tick - Cells: the basic building blocks of life - Types of polymers and their roles in biology - The languages of life: how nucleic acids and proteins communicate - DNA and RNA—what they are and what they do - What happens when genes change: understanding mutations - The difference between helpful and harmful gene mutations

What makes antisense oligonucleotides (ASOs) so special? Let’s first understand what an oligonucleotide is. An oligonucleotide is a short strand of synthetic DNA or RNA (a nucleic-acid chain), usually consisting of up to approximately 20 nucleotides long—designed to bind with specific sequences in the body. At n-Lorem, our ASO technology is built on more than 30 years of research, innovation, and investment. It’s uniquely suited for treating nano-rare diseases—ultra-rare genetic conditions that affect just one or a few individuals. The versatility and specificity of ASOs allow us to address a wide variety of gene mutations, creating customized therapies for each unique patient. Compared to other traditional drug discovery platforms, discovering and developing an optimal ASO is inexpensive, quick and can be used to treat diseases that are caused by many different types of gene mutations. ASOs work by binding to RNA, thereby modifying the expression of disease-causing proteins. This makes them exceptionally well-suited for treating diseases caused by rare or unique genetic mutations. On This Episode We Discuss:

- The repurposing of small molecule drugs

- The promise and limitations of gene replacement therapies

- What makes ASO drug development different—and better—for nano-rare diseases

- A brief history of modern drug development

- How regulatory frameworks evolved after medical disasters

- The decentralization of the biotechnology industry

- What challenges still lie ahead in genetic medicine

Intro to Medical Science Series YouTube Playlist: https://www.youtube.com/playlist?list=PLrDVyc3t26Fy5aQpo3mulackGlUwrIqYL

This episode is brought to you by Hongene Biotech!

On this episode we discuss:

2:45 – What Connor’s life was like from birth

4:20 – Finding a SCN2A diagnosis through genomic sequencing

6:40 – A hallway conversation between Kelley and Stan about Connor sparked the idea of the n-Lorem Foundation

9:20 – Dr. Olivia Kim-McManus on engaging and gaining institutional support to treat n-of-1

11:40 – Connor's program was uniquely challenging

14:18 – Connor unknowingly had coronavirus upon receiving his first ASO dose

18:00 – Symptoms that affect Connor’s life on a daily basis

18:55 – Connor has walked unassisted for the first time in his life

26:10 – Finding the optimal treatment dose and schedule

29:05 – Improvement of painful gastrointestinal (GI) issues

Bios:

Kelley Dalby is the Director of Natural History and Diagnostics in Epilepsy at Praxis Precision Medicines, where she has worked for four years. Kelley worked as a high school English teacher in San Diego until her son Connor was born with a severe form of epilepsy caused by a mutation in the SCN2A gene. When he was diagnosed, she co-founded a biotechnology company, RogCon, focused on discovering therapies for SCN2A mutations. Kelley served as Vice President and contributed to the companies’ success, including licensing the primary program to Praxis Precision Medicines.

Dr. Olivia Kim-McManus is an Associate Clinical Professor, UC San Diego School of Medicine Dept of Neurosciences, Pediatric Neurologist and Epileptologist, Rady Children’s Hospital Precision Therapeutics Neuro-Interventional Program Director. She received her undergraduate degree in Neurosciences at Columbia University in New York City and medical degree at George Washington University Children’s National Medical Center. She specializes in treating children with medically intractable epilepsy due to rare genetic etiologies requiring targeted genetic therapies or epilepsy surgery. She is the Director of the Batten’s Disease Neuro-infusion Program at Rady Children’s Hospital where she delivers intraventricular cerliponase-alfa enzyme replacement therapy via Ommaya reservoir targeted for disease modifying therapy for rare pediatric genetic disease. Dr. Kim-McManus is Neurology Section Vice Chief of Medical Staff Executive Committee at Rady Children’s Hospital, ACGME Epilepsy Fellowship Associate Program Director at UCSD, Epilepsy Foundation San Diego Professional Advisory Board member, Rady Children’s Insitute for Genomic Medicine clinical investigator, and UCSD Altman Clinical and Translational Research Institute Scientific Review Board member.

Links: n-Lorem Candle and Card Fundraiser - https://www.nlorem.org/mothers-day-candle-2025/

Hongene - https://www.hongene.com/