12 biotech unicorns revolutionizing the future of healthcare

Have you ever heard of a biotech unicorn? Although a mythological creature might be what first springs to mind, a unicorn in the business world actually refers to a privately owned company valued at more than $1 billion. Some world-renowned former unicorns include Airbnb, Facebook, and Google. The reason they are called “unicorns” is because it is traditionally very rare for a company to achieve this valuation. 

In the biotech world, there are only a select few companies with unicorn status that are working on therapeutics. In this article, we take a closer look at 12 biotech unicorns rated in order of their valuations according to CB Insights.

Abogen Biosciences

Abogen Biosciences is a Chinese biotech company and is valued at $3.7 billion. This comes as no surprise after it raised $700 million in series C funding back in August 2021, marking one of the largest private biotech funding rounds ever and obliterating the fundraising record for a Chinese mRNA vaccine developer. It then followed this round with another impressive series C+ round in November of the same year, in which it raised $300 million.  

Abogen is part of a rare breed of Chinese biotechs, having actually developed all of its key messenger RNA (mRNA) platform technologies itself, across mRNA synthesis, delivery, and manufacturing. Its mission is to develop therapies derived from mRNA technology, including immunotherapies for cancer, protein replacement therapies, and a vaccine against COVID-19. The vaccine is the company’s lead candidate and was granted emergency use authorization in Indonesia in 2022, making it the first China-made mRNA vaccine authorized for use.  

Eikon Therapeutics

A drug discovery and development startup helmed by former Merck research chief Roger Perlmutter, Eikon Therapeutics is valued at $3.2 billion. The company’s latest fundraising round was in June last year, in which it raised nearly $106 million in series C equity financing, bringing its total raised since its public debut in 2021 to nearly $775 million. 

The biotech unicorn was formed based on a Nobel Prize-winning technology that allows scientists to look at how proteins move inside of cells. As well as developing some drugs in-house, Eikon has also acquired programs from other companies; in 2023, it gained the rights to several drug candidates, including two clinical-stage toll-like receptor 7 and 8 agonist immune modulators from Seven and Eight Biopharma for the treatment of cancer, two clinical-stage PARP inhibitors from Impact Therapeutics, and “a suite of preclinical assets” from Cleave Therapeutics that address targets involved in areas that have importance in cancer and in neurodegenerative disease.

insitro 

Despite being one of the most well-funded artificial intelligence (AI) players in the biotech industry since it was founded in 2018, having raised more than $700 million in capital to date, insitro has managed to keep a relatively low profile over the last few years. But it is fair to say that this has not had an impact on the company’s worth, as it is valued at $2.44 billion. The company’s mission is essentially to integrate multimodal data from human cohorts and cellular models with the power of AI and machine learning (ML) and uncover genetic targets and new therapeutic hypotheses, leveraging human and cell data to increase the probability of success. 

Just recently, insitro came out of its “quiet mode” to announce that it had entered into three strategic agreements with Eli Lily focused on advancing potential new medicines for metabolic diseases, including metabolic dysfunction-associated steatotic liver disease (MASLD), based on targets identified by insitro using its AI-based platform. In addition to metabolic diseases, the biotech unicorn is also working in neurodegeneration and oncology.

Xaira Therapeutics

Xaira Therapeutics only launched in April 2024, but armed with $1 billion in financing, the young startup is already considered a biotech unicorn and is valued at $2.15 billion. The company is combining machine learning, data generation, and therapeutic product development to build a platform for drug discovery that will advance multiple drug programs and unlock biological understanding to inform future discovery.

Part of Xaira’s team includes researchers who developed models for protein and antibody design called RFdiffusion and RFantibody in Baker’s lab. The company will use these models to develop new ways to connect biological targets and engineered molecules to human diseases.

Cambrian BioPharma 

Cambrian BioPharma is valued at $1.79 billion. The company closed a series C round in 2021 worth $100 million, bringing its total raised to $160 million since its inception in 2019. More recently, in 2023, the company launched a new pipeline company called Amplifier Therapeutics to boost its anti-aging pipeline, as Cambrian’s mission is to develop therapeutics to lengthen the period of life spent in good health.

Cambrian operates as a Distributed Development Company (or DisCo), which is ultimately designed to bridge the gap that exists between academic discovery and drug development. This model combines the advantages of a venture capital firm and a big pharmaceutical company, with the nimbleness of a biotech startup. The company’s sourcing and development engine enables the identification of promising science, deep due diligence, deployment of capital and teams of drug development experts all under the same roof. 

Tessera Therapeutics 

Having made headlines in April 2022 after raising $300 million in series C financing, Boston-based Tessera Therapeutics is now valued at $1.69 billion. The proceeds from the series C round have helped to fund the development of the company’s Gene Writing technology, which can make a range of edits and insertions into the genome to deliver therapeutic genes.

Tessera’s Gene Writing technology is based on a different concept to CRISPR called mobile genetic elements (MGEs). These are a vast group of genomic sequences, including plasmids and transposons, that are able to move within a genome or jump to other organisms. The technology uses RNA templates to make small edits to genetic code or DNA templates to insert whole sequences. The technology has the potential to cure genetic diseases, even those that current gene editing technology cannot. 

Orna Therapeutics 

Orna Therapeutics is a leader in the circular RNA field and is currently valued at $1.5 billion. The biotech unicorn launched in 2021 with $100 million in series A financing, before securing another $221 million in series B financing a year later in August 2022. Around the same time as this second round, Orna also entered into a collaboration with Merck to discover, develop, and commercialize multiple programs, including vaccines and therapeutics in the areas of infectious disease and oncology. Most recently, the company also acquired ReNAgade Therapeutics in an attempt to solidify its leadership in the circular RNA field.

Circular RNA is a form of single-stranded, non-coding RNA that forms a covalently closed continuous loop, meaning that the 3’ and 5’ ends that are usually present in an RNA molecule are joined together. Orna’s approach includes two delivery platforms: panCAR, which targets multiple lineages of the immune system at once, and SiTu Editing in the Marrow (STEM), which targets stem cells in the bone marrow. The company is still in the preclinical stages and its pipeline includes programs in oncology, autoimmune, genetic, and other diseases. 

Generate:Biomedicines 

With a valuation of $1.36 billion, Flagship Pioneering company Generate:Biomedicines is using AI to create new, innovative drug candidates. Just last month, the company partnered with pharma giant Novartis in one of the biggest biotech deals of September 2024. The two companies are collaborating to discover and develop protein therapeutics across multiple disease areas with the help of Generate’s generative AI platform. The deal saw Generate receive $65 million upfront, with the potential to bag up to $1 billion in milestone payments. And, looking further back to September 2023, Generate managed to raise $273 million in a series C financing round, which was the largest private biotech investment of September 2023. This capital brought the total raised by the company since 2020 to nearly $700 million.

Since the company’s series B financing round in 2021, Generate:Biomedicines has made notable progress as a leader in the field of generative biology; it has initiated its first-in-human trial for GB-0669 – a monoclonal antibody targeting a highly conserved region of the spike protein –in SARS-CoV‑2, entered into multiple high-profile collaborations, and expanded The Generate Platform into new modalities, including into bispecifics, enzymes, T‑cell engagers, and cell therapies. The company now has a robust pipeline of 17 programs in preclinical and clinical development across oncology, immunology, and infectious disease.

Kallyope 

New York-based biotech Kallyope is valued at $1.22 billion and is focused on developing oral weight loss medications tailored for patients dealing with obesity, diabetes, and gastrointestinal disorders. Its latest funding round was in 2022, in which it bagged $236 million in series D financing. More recently, in 2023, the company also received an $8.2 million grant from the Bill & Melinda Gates Foundation to identify promising bioactive compounds for improving maternal, newborn, and children’s health in the epicenters of the global food and nutrition crisis. 

Kallyope has a diverse portfolio of novel, oral small-molecule therapies across the aforementioned disease areas. The biotech unicorn initiated a phase 2 trial last year evaluating two of its assets – K-757 and K-833 – for the treatment of obesity and type 2 diabetes. The trial is aimed at demonstrating the weight loss efficacy, safety, and tolerability of these drug candidates, which are oral nutrient receptor agonists that enhance the body’s natural metabolic signals to stimulate the secretion of appetite-suppressing satiety (the state of feeling full) hormones, including GLP-1 and several other well-validated satiety hormones.

IntraBio

With a valuation of just over $1 billion, neurological disease company IntraBio reached unicorn status thanks to its latest funding round in March 2024, in which it secured more than $40 million. Around the same time, the U.K. biotech also decided to move its corporate headquarters to Austin, Texas in the U.S. 

To top things off, the company’s lead candidate received U.S. Food and Drug Administration (FDA) approval late last month for the treatment of the rare lysosomal storage disorder Niemann-Pick disease type C. Sold under the brand name Aqneursa, the oral treatment is a modified amino acid drug that works by targeting and correcting the genetic mutations responsible for Niemann-Pick disease, helping to reduce lipid accumulation in cells and slow disease progression. The company’s current pipeline has broad applicability to be developed as novel treatments for both rare and common neurological disorders.

Mammoth Biosciences 

CRISPR-focused biotech Mammoth Biosciences officially achieved unicorn status after it raised $195 million back in 2021, bringing its valuation to just over $1 billion. The company is leveraging its proprietary CRISPR platform for both therapeutics and diagnostics and is focused on the discovery and engineering of novel CRISPR systems, particularly Cas14 and CasΦ (phi) enzymes, which are significantly smaller and have increased temperature stability, as well as a faster reaction that enhances their effectiveness in vivo genome editing and diagnostics. 

Mammoth’s pipeline is still in the preclinical and research stages, with the indications of its candidates mostly undisclosed. Moreover, the company recently signed a partnership deal with Regeneron that is worth $100 million upfront plus $370 million in milestones per target. The collaboration is aimed at researching, developing and commercializing in vivo CRISPR-based gene editing therapies for multiple tissues and cell types.

Owkin

Also valued at $1 billion, French company Owkin is known for its AI-based approaches to identifying new treatments, optimizing clinical trials, and developing diagnostics, with a primary focus on oncology. The company reached unicorn status after Sanofi invested $180 million into the AI company in 2021. This was also part of a broader strategic alliance between the two companies aimed at refining clinical trials and identifying new biomarkers for various cancers, including lung, breast, and multiple myeloma​​. The partnership was recently expanded to include immunology. 

The biotech unicorn has also entered into collaborations with Bristol Myers Squibb and Merck. Furthermore, Owkin launched the PortrAIt project last year, which is a €33 million ($36 million) AI-powered precision medicine venture for cancer diagnosis and treatment, with backing from the French Government. The project seeks to establish a research and collaboration platform, with the aim of implementing new AI-based patient diagnostics in French hospitals within four years​​.

Biotech unicorns: A rare breed

As of May 2024, there are more than 1,200 unicorns around the world. However, it’s worth noting that unicorns in the biotech industry are not valued as highly as in other industries, such as enterprise technology, media and entertainment, and even industrials. For example, OpenAI is valued at $157 billion according to CB Insights, and ByteDance is valued at $225 billion. Comparatively, the highest-valued biotech unicorn, as we can see from this listing, is only worth $3.7 billion.