Top 10 most promising biotech startups to watch in 2021

Digital health products are changing the way biotech startups are evolving. With all their innovative tools and technologies digital health is switching the paradigm for biotech startups who work on healthcare devices - from the initial product idea and up to the actual development. The digital health industry’s potential pushes biotechnology to come up with more effective innovations, extend business boundaries, and improve relationships with potential patients.

A constantly changing situation in the healthcare sector promises risks, but at the same time, high profits for investors. So, which of the most promising biotech startups could change the world? Let's figure it out

Development of drugs for central neural system diseases

In this section, we'll look at three leading startups that are improving therapies for various neurological illnesses. These diseases affect millions of people around the world. Hope for a breakthrough in science remains as we wait for the end of clinical trials for Cerevel Therapeutics, Cyclerion Therapeutics, and Passage Bio.

Cerevel Therapeutics. According to the top biotech startups 2020 ranking, Cerevel Therapeutics (Massachusetts) took first place with 45 points. Bain Capital Private Equity and Bain Capital Life Sciences have invested about $350 million in this project, and Pfizer helped to launch it. They want to develop medicines to prevent and treat Parkinson's disease, Alzheimer's disease, and epilepsy. Currently, the company is in the first stage of clinical trials for psychosis treatments, and in the second stage - for finding a progressive medication for neurodegenerative diseases. In clinical trials, Lorazepam has shown itself well in epilepsy treatment.

Cyclerion Therapeutics. Among top biotech startups to watch, Cyclerion Therapeutics (Massachusetts), with a $175 million budget, takes a special place.

In the brain, signaling occurs via the NO-cGMP pathway. Cyclerion Therapeutics concluded that dysfunctions in this pathway lead to neurodegenerative diseases (Alzheimer's and schizophrenia). For a person to be healthy, it is necessary to strengthen this pathway. That is how the allosteric modulator CY6463 was found: it can enhance NO signaling. Preclinical animal tests showed that memory, spinal density, and cerebral blood flow have improved, and gene expression restored in people with such diseases after experimental treatment.

Passage Bio (Pennsylvania) is another company among the top 10 biotech startups specializing in rare monogenic diseases of the central nervous system. They are focused on developing transformative gene therapies to address and modify them. Several large companies funded Passage Bio for $335.5 million in 2019. They develop treatments for GM1 gangliosides, address the causes of frontotemporal dementia, and treat Krabbe disease.

Cardiovascular diseases prevention

Cardiovascular diseases are a common cause of death worldwide. Because most people are living in polluted areas, eat unhealthy food, smoke, drink alcohol, and, for instance, take warfarin, a blood-thinning remedy, they put themselves at big risk. About 23.6 million people are forecast to die from cardiovascular diseases in 2030. It means that scientific and technological progress must advance faster and thus save more lives.

Anthos Therapeutic, a clinical-stage biopharma company, develops and commercializes therapies for high-risk cardiovascular patients. About one in four death worldwide account for thrombotic disorders. There is still an unsatisfied need for safe antithrombotic remedies that can be used for patients that are currently underserved and can’t take traditional anticoagulants, so the company obtained a license to Anthos MAA868 from Novartis. It’s an anti-thrombotic that can potentially prevent many cardiovascular disorders with minimal or no bleeding risks. The therapy works well with other drugs.

Cancer treatment

According to WHO forecasts, the number of cancer patients worldwide will grow by 60%. Here are the companies who try to find a cure for the disease.

Century Therapeutics. Among biotech oncology startups, Century Therapeutics (Pennsylvania) stands out for using induced pluripotent stem cells (iPSC) to create genetically engineered and immune cell products with the potential to become new cell therapies. These effector cells, obtained from healthy adults, can target different types of tumors. A specific genome reprograms them. The cells can differentiate into some types of immune cells and are then used to create drugs.

Thrive and Ribon Therapeutics. Other biotech startups we’re going to talk about are Thrive and Ribon Therapeutics (Massachusetts), with $110 million and $95 million of funding, respectively. They have launched projects to diagnose and treat cancer and are known to be the best biotech startups to work for.

Thrive is aspired to create a future where cancer diagnostics include simple blood tests. So it's gaining momentum with the CancerSEEK biopsy test, which will detect cancer in the early stages. It's designed to become a simplified complementary screening option. The company has also developed the Thrive Early Detection platform, which aims to interpret the results as follow-up testing to confirm the diagnosis.

Ribon Therapeutics is trying to treat advanced tumors while using specific medicine, a drug called MonoPARP that slows down the metabolic activity of cancer cells. It forces them to stop growing and dividing. The antitumor immune response becomes active, which allows fighting large tumors.

Medicines for rare diseases

Today, a large number of startups are aimed at helping with rare diseases. They show great promise to use their drugs in clinical practice. This section focuses on two unique companies that have created truly great projects. Mirum Pharmaceuticals aims to treat liver diseases. It also gives a chance to live for those who won't have a liver transplant. Maze Therapeutics is also a promising company looking for a cure for the disease through genetics.

Mirum Pharmaceuticals. One of Silicon Valley's top biotech startups is Mirum Pharmaceuticals (California), with $195 million. They provided hope for people with rare liver ailments including Alagia syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). Mirum has already shown a positive effect of maralixibat in comparison with available therapy. The drug is a liquid for oral administration. It works by blocking the transport protein of bile acids, thereby lowering their levels.

PFIC is a progressive disease that requires liver transplantation. The cells are unable to excrete bile, which leads to its accumulation. It causes constant severe itch, the body's inability to grow and develop. Jaundice often accompanies PFIC and can be fatal if there won't be transplantation. In the United States, people manage to get only 600 transplants per year. Most children don't have a chance to survive.

The company launched the 3-rd phase of clinical drug trials in children 1 to 17 years old. More than 119 children and 69 adults have received maralixibat therapy. 1.400 patients also took the remedy in other studies. An experimental treatment with INDIGO confirmed that the drug is a breakthrough therapy at the 2nd phase. In comparison with all available treatment methods, it shows a health improvement.

Maze Therapeutics. Also, Maze Therapeutics (California) can boast of the top biotech startups in the US. The research attracted several large investors with a total capital of $191 million. Their goal is to spell out the causes of severe diseases at the genetic level while using advanced algorithms and data science. The idea of a startup is about different manifestations of gene mutations. For example, close relatives who have a disease-causing gene may feel differently. Up to the point that one of them may not have a dangerous illness. To cure the sickness, it's necessary to understand its origin and manifestation. Researchers are struggling with the issue of finding genetic modifiers that prevent genes from manifesting.

Treatment of infectious diseases

In the statistics for 2019, WHO submitted data that 2.6 million people died from the upper respiratory tract's infectious diseases.

Qpex Biopharma. The COVID-19 pandemic has reminded humanity that an incurable infection can spread quickly and remain untreatable for a critical time. Antimicrobial drug resistance is another pandemic: it much slower, but it is, also, a force to be reckoned with. Qpex Biopharma finds new chemical classes of drugs that can overcome drug resistance, reserve antibiotic resistance, improve their efficiency, and, in general, discover, develop, and approve new anti-infective medicines. 

innovative scientific companies

Laboratory grown meat

Yes, the media have already made a splash about this project all over the world. Memphis Meats (California) develops animal proteins that don't require traditional agricultural methods. You can now eat chicken, duck, or beef without killing animals. Cell culture technology preserves the regular meat qualities, making the product even more nutritious and safe. That will significantly reduce industrial space and greenhouse gas emissions. So the planet can be greened and cleaned of chemical production waste. Today the company is overcoming the so-called “legal hole”. It slows down the meat market entry. Additional investment is needed to help with making the product ubiquitous.

Accelerating crop growth

Indigo Agriculture (Massachusetts) has developed technology to increase crop yields. Special biotrinsic microorganisms coat the seeds. They help to resist weeds and pests. They also improve survival in dry and poor soils, protecting crops from stress. Interestingly, the biotrinsic microbes themselves were isolated from plant tissues that grew under extreme stress conditions. It was these bacteria that became the defense mechanism that allowed them to survive. So each biotrinsic preparation is adapted to a specific culture type. Currently, the indicators in the biomass growth of cotton, soybeans, rice, and wheat have improved. Indigo says plants will grow 40-70% faster.

The company has several branches: an effective Marketplace to sell products more efficiently and a logistics platform called Indigo Transport. Indigo Carbon is sponsoring investment in soil enrichment, helping farmers transition to more profitable practices.

Genome editing

Beam Therapeutics (Massachusetts) has presented an innovative genetic code correction project which has been successfully applied in many laboratories. CRISPR technology allows you to cut out unnecessary DNA fragments with special molecular scissors. The ends are connected non-homologically to fill the gap in the sequence. That is how the genetic information changes and a mutation occurs in a known area. The company also created a technology that allows changing the nucleotides (adenine, guanine, thymine, cytosine, uracil) without excision. We can see it launched in the next few years.

Tissue regeneration

Samumed is one of the richest and most famous startups. It's focused on solving vital problems such as hair loss, preventing fragility of bones and joints, skin ailments, and the aging process reversing. In its research, the company pays special attention to stem cells. The first clinical studies showed that the medicines are quite safe. They positively affected joint mobility in arthritis, stimulated hair growth, and also reduced pain. The company has already presented the phase 2 data and the results sound very promising for the ones suffering from osteoarthritis.

Pandorum Technologies have ventured to breathe life into the bioengineered eye cornea of a human. This might seem surreal to a common person, but not only for the two enthusiastic co-founders of this biotech startup. They are working on the cornea blindness treatment and willing to make it as perfect and fast as possible. Pandorum Technologies’ “Bioengineered Liquid Cornea” is one of the options for people with trouble seeing to restore their vision. The material acts as a matrix that helps to accelerate human tissue regeneration. We can’t help mentioning that this method of vision restoration is completely a scarless process that involves tissue regeneration. The bioactive components of the liquid cornea are programmed to activate scarless wound healing. And that makes this startup even more prominent, as the treatment appears to be non-invasive.


Microsoft CEO Satya Nadella noted that in just two months, technological progress has advanced two years ahead. 2020 has opened up new funding and growth opportunities for many top biotech startups. Truly unique things create a buzz around them that gives a chance for generous funding. Unfortunately, some innovations aren't always attracting media attention and may disappear from the market forever.

Yes, risks are expensive, but they allow you to move forward and discover new technologies. Developers are the progress creators, but without ambitious entrepreneurs, talented leaders, and investors who believe in success, the development would be impossible. All of these people are working on breakthroughs in science and prioritizing saving and improving human life.

All the modern technologies for medicine, tissue regeneration, drug creation for rare diseases and cancer treatment, improvement of crops, and genetic code correction are sometimes beyond our imagination. It seems that we're living precisely in that era when all these inventions are finding the attention they deserve.


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