November 11, 2024
This is the final Biotech-20 list.
- ACLX – Arcellx Inc: A young biotech company focused on developing engineered CAR-T cell therapies for cancers such as multiple myeloma, aiming to enhance CAR-T efficacy and safety.
- ALNY – Alnylam Pharmaceuticals Inc: A leading RNAi therapeutics company developing treatments based on RNA interference technology, with a focus on genetic diseases, cardiometabolic disorders, and rare diseases.
- AMGN – Amgen Inc: A biotechnology company that develops and manufactures innovative human therapeutics, focusing on areas like oncology, cardiovascular health, and inflammatory diseases.
- AXSM – Axsome Therapeutics Inc: A biopharmaceutical company developing therapies for central nervous system (CNS) disorders, including depression, migraines, and Alzheimer’s disease-related agitation.
- BNTX—BioNTech SE ADR: Known for its COVID-19 vaccine, BioNTech develops immunotherapies based on mRNA technology, which have applications in infectious diseases and cancer.
- CRSP – CRISPR Therapeutics AG: A gene-editing company focused on creating transformative gene-based medicines for genetic diseases, utilizing CRISPR/Cas9 technology.
- GILD – Gilead Sciences, Inc: A biopharmaceutical company specializing in antiviral drugs, especially for HIV/AIDS, hepatitis B, hepatitis C, and oncology.
- ILMN – Illumina Inc: A company that develops, manufactures, and markets integrated systems to analyze genetic variation and function, primarily through sequencing and array-based technologies.
- IONS – Ionis Pharmaceuticals Inc: A biotechnology company pioneering RNA-targeted drug discovery and development to treat diseases with high unmet needs, including neurological and rare diseases.
- MRNA – Moderna Inc: A biotech company known for its mRNA-based COVID-19 vaccine, Moderna focuses on messenger RNA therapeutics and vaccines for infectious diseases, cancer, and other conditions.
- NTLA – Intellia Therapeutics Inc: A biotechnology company focused on CRISPR/Cas9 gene-editing technology to develop curative therapies for genetic diseases and cancer.
- REGN – Regeneron Pharmaceuticals, Inc: A biopharma company specializing in discovering and developing antibody-based treatments for conditions such as cancer, eye diseases, and allergic diseases.
- RGLS – Regulus Therapeutics Inc: A biopharmaceutical company focused on developing microRNA therapies for treating liver and kidney diseases.
- RGNX – Regenxbio Inc: A biotechnology company specializing in gene therapy treatments for retinal, neurodegenerative, and metabolic diseases.
- SMMT – Summit Therapeutics Inc: A biopharmaceutical company focused on developing antibiotics for infectious diseases, particularly addressing antibiotic-resistant bacterial infections.
- TGTX – TG Therapeutics Inc: A company dedicated to developing novel treatments for B-cell malignancies and autoimmune diseases, emphasizing therapies for multiple sclerosis and hematologic cancers.
- TIL – Instil Bio Inc: A biotech company focused on developing T-cell therapies for solid tumors, aiming to improve the effectiveness of cell-based cancer treatments.
- VKTX – Viking Therapeutics Inc: A clinical-stage biopharmaceutical company focused on metabolic and endocrine disorders, including treatments for liver disease and obesity.
- VTYX – Ventyx Biosciences Inc: A biotechnology company developing therapies for autoimmune and inflammatory diseases, particularly in dermatology, gastroenterology, and rheumatology.
- WVE – Wave Life Sciences Ltd: A genetic medicines company using stereopure oligonucleotides to develop treatments for genetic disorders, focusing on rare diseases and neurology.
I replaced Rigetti Computing (RGTI) with Alnylam Pharmaceuticals (ALNY) and IonQ (IONQ) with Arcellx Inc. (ACLX) from the list I published last week.
Rigetti Computing was out of place because it’s not a biotech or pharmaceutical company. Unlike the other companies, which focus on drug development, gene therapy, and healthcare, Rigetti is focused on quantum computing technology. Rigetti’s primary mission is to advance quantum computing hardware and software in fields like AI, material science, and cybersecurity. While quantum computing has potential uses in healthcare — such as accelerating drug discovery and analyzing complex biological data — Rigetti itself is not directly involved in developing biotech or pharmaceutical products, so inclusion on this list did not meet our objective to focus strictly on companies within the biotech or pharmaceutical sectors.
I replaced IonQ (IONQ) with Arcellx Inc. (ACLX) for the same reason.