biotech exam 1

- GA Department of Agriculture and US Department of Agriculture and Plant Health Inspection Service (USDA - APHIS) found Highly Pathogenic Avian Influenza (HPAI)

a commercial poultry operation

- the 1st confirmed case in 2026
- 4th case at the location since Feb 2022

- depopulation
- disposal
- disinfection
- 10 km quarantine
- surveillance

number of common shares multiplied by the current price of those shares.

market cap below US$1 billion

market cap between US$1 billion and US$5 billion

market cap exceeds US$5 billion

far more controversial

market cap under US$100 million

market cap under US$50 million

sometimes used as a synonym for a large-cap

penny stock, regardless of share price

- return over recent life of stock
- the single most consistent red flag to excessive optimism and over-investment
- a marker of business problems and opportunities

how cheap or expensive a stock's shares are.

- earnings multiple
- multiple
- P/E
- PE

less you have to pay for the stock, relative to what you can expect to earn from it.

price per share (numerator): the market price of a single share of the stock.

earnings per share (denominator): net income of the company for the most recent 12-month period, divided by number of shares outstanding.

24/3=8
- you are paying $8 for every one dollar of earnings

less than 20-25

analyze the market's valuation of a company's shares relative to the wealth the company is actually creating

1. does a company have a drug that will complete phase 3 clinical trials soon?
2. does a company show an increase in its stock price over the short term?
3. does a company have an IPO soon

• Idea
• Provisional patent filed (NO public disclosures) need a patent attorney
• Patent filed -- years go by....
• Company is formed and registered
• What is the size of the potential market?????
• Show me the money....Investors!
• Angel investors; Venture capitalists
• Investment funds...Foundations
• Rounds of investment
• Odds of success? < 10% but better than Vegas!

SBIR/STTR from NIH
• Georgia Research Alliance Venture Fund
• State-funded grants (like CPRIT)
• Angel investors
• Foundations and their Venture Funds
• Money needed to fund clinical trials

Indicates cheaper stock relative to earnings.

High P/E may indicate over-optimism or over-investment.

851.

~\$7.51 trillion.

Vivek Ramaswamy.

Studied biology at Harvard, worked at hedge funds and Goldman Sachs, attended Yale Law School.

Holding company founded by Ramaswamy.

$2.2 billion.

Alzheimer’s drug candidate purchased for \$5 million.

Over \$38 million in capital gains in IPO year.

Inflating asset value before failure or sell-off.

Failed clinical trial two years later.

High-impact, breakthrough therapy addressing unmet medical needs using novel technology.

Gene-editing treatment for sickle cell disease.

Biotech company focused on carbohydrate-based therapeutics.

Drug class targeting selectins involved in inflammation and sickle cell disease.

Glycomimetics’ pan-selectin antagonist partnered with Pfizer in 2011.

Glycomimetic drug for sickle cell disease that failed Phase 3 trial in 2019.

Missed primary endpoint of readiness-for-discharge.

Second flagship product targeting AML.

Discontinued after Phase 3 failure in May 2024.

\$880 million.

\$17.66 million.

Original Glycomimetics stock ticker.

Merged with Crescent Biopharma on June 16, 2025.

Crescent Biopharma, Inc.

CBIO.

GLYC shareholders received 1:100 reverse split.

Glycomimetics shareholders own ~3% of new company.

DNA → RNA → Protein.

Proposed the central dogma circa 1965.

International effort to sequence the human genome.

15 years.

13 years.

~\$3 billion total; ~\$400 million sequencing.

Near-complete high-quality human genome sequence.

Identify all genes and regulatory elements.

Permanent record of genetic information.

Provides framework to study genetic basis of disease.

Dideoxy chain termination method developed in 1977.

Enable automated Sanger sequencing.

Nobel Prize winner (1980).

~500–800 bases.

~3 billion base pairs.

~130 Mb.

Yeast Artificial Chromosome for cloning large DNA fragments.

Overlapping clones forming continuous DNA regions.

Random fragmentation and computational reassembly of DNA.

Number of times a base is read during sequencing.

≥30× for accuracy.

Coverage compared to overlapping raindrops on sidewalk.

Director of public HGP.

Founder of Celera Genomics.

HGP strategy.

Celera strategy.

February 2001.

Gene-rich, transcriptionally active (~92%).

Repetitive, tightly packed (~8%).

Completed full human genome in 2022.

High-throughput parallel sequencing platforms.

Computing power doubles every ~2 years.

~\$600 per human genome.

Leading sequencing company headquartered in San Diego, CA.

~\$22 billion.

Uses reversible terminators and bridge amplification.

HiSeq, MiSeq, NovaSeq.

Single-molecule, long-read sequencing.

Long-read sequencing company.

Portable sequencing technology (MinION).

Generates 350 Gb/day; ~\$500enome.

Single-nucleotide polymorphism.

~1 per 300 bases in humans.

Humans differ by ~0.1%.

International catalog of human genetic variation.

2,504 individuals from 26 populations.

84.7M SNPs, 3.6M indels, 60k structural variants.

Tailoring treatment based on genetic variation.

2016 legislation funding NIH initiatives.

NIH program with >1M participants.

~250,000.

>1 billion variants (Nature 2024).

Serine protease regulating LDL receptor recycling.

Lower LDL and reduced CAD risk.

Elevated LDL and increased CAD risk.

Alirocumab, Evolocumab (Repatha).

Genome-Wide Association Study.

Identify genetic variants associated with traits/disease.

Arrays analyzing millions of SNPs.

Grants negative right to exclude others from using invention.

Utility, design, plant.

Diagnostics, compositions of matter, functional uses.

Supreme Court ruled GM bacteria patentable.

Novelty, inventiveness, industrial application.

Purified DNA deemed patentable.

Sequences must have specific, substantial utility.

Tumor suppressor genes linked to breast/ovarian cancer.

Up to 85% lifetime risk by age 70.

Company holding BRCA patents.

\$3,700.

~\$5,000.

Naturally occurring genes not patentable.

Patentable due to human modification.

Increased trade secrets and diagnostic competition.

Protects against genetic discrimination.

Regulates medical genetic data privacy.

1 in 400 women carry mutation.