Keep an eye on these 15 biotech companies in 2026 

For this year’s ‘biotechs to watch’ selection, each Labiotech journalist picked five companies they believe will be worth following in 2026. Some earned their spot after clear momentum in 2025, while others have important clinical, regulatory, or strategic milestones ahead in the coming year. The result is a list of emerging biotech and more established players. This is a snapshot of the biotech companies that stood out to our editorial team as we head into 2026.  

AAVantgarde Bio 

The Milan-based biotech company is built around two ways of getting oversized genes into cells: a dual-hybrid AAV approach at the DNA level and an intein-based system that reassembles proteins inside the cell. Those technologies are being tested first in Usher syndrome type 1B-associated retinitis pigmentosa with AAVB-081 and in Stargardt disease with AAVB-039, both conditions driven by genes too large for standard AAV vectors. 

In 2025, both programs moved firmly into the clinic. LUCE-1, the phase 1/2 study of AAVB-081 in Usher 1B, continued to enroll, with updated data presented at EURetina 2025 and an ongoing focus on safety and early signals of visual function in a population facing combined deafness and progressive blindness. For Stargardt, AAVB-039 received IND clearance in mid-2025 and U.S. Food and Drug Administration (FDA) fast track designation in August, and the CELESTE phase 1/2 trial is now running. The company then added financial depth with a $141 million series B in November. 

In 2026, AAVantgarde aims to reach clinical proof-of-concept in both lead indications. Because both targets, MYO7A in Usher 1B and ABCA4 in Stargardt, have a history of difficult development, the early human data from LUCE-1 and CELESTE will be watched in 2026. 

Abivax 

Abivax entered 2025 as a company with promise; it ended the year as one of Europe’s standout biotechs. The turning point came with the two phase 3 trials in ulcerative colitis, where obefazimod consistently delivered: both studies met their primary and key secondary endpoints. The strong clinical readout triggered a surge of more than 500% in Abivax’s share price and helped lift its valuation above €3.5 billion ($4.11 billion). With a subsequent $747.5 million public offering, the company secured the financial runway to move from late-stage development toward registration. 

Indeed, the company’s stock became one of the standout biotech performers of 2025, a trajectory that has also fueled ongoing market speculation about interest from larger pharmaceutical players, including Eli Lilly. 

The next 12 months will show how far Abivax can push this momentum. The 44-week ABTECT maintenance trial is expected to read out in the second quarter of 2026, with regulatory filings planned shortly after if the data hold up. At the same time, the company is moving obefazimod beyond ulcerative colitis: induction data from the ENHANCE-CD program in Crohn’s disease are anticipated in the second half of the year. 

Aspen Neuroscience 

Regenerative therapies represent a paradigm shift in medicine, as they aim to repair damaged tissues instead of just behaving as band-aids to manage symptoms. California-based Aspen Neuroscience is working towards this with its induced pluripotent stem cell (iPSC)-derived autologous cell replacement therapies for patients with Parkinson’s disease who have unmet medical needs. 

Using the patient’s own cells, Aspen’s technology is designed to restore dopamine function, which is lost in the case of Parkinson’s disease as the neurons producing dopamine die, leading to symptoms that affect movement, such as tremors, stiffness, slowness, and balance issues. By rebuilding neural networks with a patient’s own cells, this minimizes the risk of the cells being rejected by the patient’s body.  

The patient’s skin cells are first retrieved and engineered into iPSCs, and then into dopaminergic neuronal precursor cells. These cells develop into dopamine-producing neurons. The American biotech’s lead candidate ANPD001, is made up of these cells, and is being evaluated in phase 1/2 trials. 

To accelerate ANPD001’s development in the clinic, Aspen raised $115 million in a series C round last month, and it is speculated that the biotech company will go public in 2026 in efforts to bring its stem cell-derived therapy to the market.  

It is a step closer to this goal, as it began enrolling its patients to test a new formulation of the candidate in September. This being the third cohort of the ongoing study, in previous cohorts, the drug was found to be safe, and patients did not need to be on immunosuppressants – medicines often administered to prevent immune systems from attacking themselves – signaling its safety. 

Beam Therapeutics 

Beam Therapeutics is undoubtedly a frontrunner when it comes to base editing – a newer and potentially safer gene editing approach that uses components from CRISPR systems together with other enzymes to directly install point mutations into cellular DNA or RNA without making double-stranded DNA breaks. In fact, in 2023, the company was the first to treat a patient in the clinic with a base editing candidate in the U.S. 

Its base editing candidates are still going strong to this day, particularly BEAM-302 for the treatment of alpha-1 antitrypsin deficiency (AATD), and risto-cel (formerly BEAM-101) for the treatment of sickle cell disease.  

In a press release in November, John Evans, chief executive officer (CEO) at Beam, stated that the BEAM-302 AATD program is a top priority for the company. BEAM-302 is currently being tested in a phase 1/2 trial; back in March, the company announced that the candidate had demonstrated the “first ever” clinical genetic correction of a disease-causing mutation in AATD, a genetic disorder where the body doesn’t produce enough of the alpha-1 antitrypsin (AAT) protein, which normally protects the lungs and liver from damage. Beam expects to provide a broad update on the program in early 2026 with new clinical data and next steps to advance the medicine to patients.  

Meanwhile, the biotech company also reported updated data from its phase 1/2 trial of risto-cel in December at the American Society of Hematology (ASH) Annual Meeting that was consistent with previously presented data. The candidate, which is being investigated in patients with sickle cell disease with severe vaso-occlusive crises (VOCs), showed positive results such as efficient and predictable cell collection, manufacturing, and release, rapid engraftment, no severe VOCs, and durable, high editing efficiency.  

Braveheart Bio 

Just last month, Braveheart Bio launched with a mission to become a frontrunner in the heart disease space, armed with $185 million in series A funding and several experienced leaders in its ranks. With its lead asset, the biotech company has what could be a rival to Bristol Myers Squibb’s Camzyos, the first and only drug to be approved for the “obstructive” form of the progressive heart condition hypertrophic cardiomyopathy.  

The company’s small molecule candidate, BHB-1893, is a selective cardiac myosin inhibitor licensed from Chinese company Jiangsu Hengrui Pharmaceuticals that is engineered to improve heart performance in patients with hypertrophic cardiomyopathy. The candidate has already undergone extensive clinical testing, including a phase 2 study in symptomatic obstructive hypertrophic cardiomyopathy, an ongoing phase 2 study in non-obstructive hypertrophic cardiomyopathy, multiple clinical pharmacology studies including a bridging study in Australia, and an ongoing phase 3 study in obstructive hypertrophic cardiomyopathy in China. 

Furthermore, early data for the drug in obstructive hypertrophic cardiomyopathy were recently presented at the European Society of Cardiology Congress 2025, demonstrating rapid and clinically meaningful reduction in left ventricular outflow tract gradients (a blood pressure measure that can speak to the severity of a hypertrophic cardiomyopathy obstruction) within days of treatment initiation, and a safety profile supporting a simple dosing regimen. Braveheart now plans to initiate late-stage clinical development of the drug in 2026, with the goal of establishing a new standard of care.

Hope Medicine 

Around 10% of women between the ages of 15 and 49 are affected by endometriosis worldwide, according to a report by the World Health Organization. Current standards of care involve hormonal therapies to quell symptoms and treatments to manage pain, but none that target the root causes of the disease. Hope Medicine seeks to change that. Located in the Chinese city of Shanghai, Hope Medicine spun out of Peking University in 2018, after which it gained the licensing rights to develop pharma giant Bayer’s then monoclonal antibody to address endometriosis. 

Dubbed HMI-115, the antibody blocks the binding of the hormone prolactin – associated with breast development and milk production – to its receptor. When prolactin binds to its receptor, it activates pathways that, in the case of endometriosis, result in the abnormal proliferation of cells outside the uterus. HMI-115 also bypasses sex hormones, such as estrogen and progesterone – hormonal treatments to alleviate symptoms – help avoid disrupting the menstrual cycle. 

The candidate was awarded fast track designation by the U.S. Food and Drug Administration (FDA) last week based on promising phase 2 results in endometriosis. Menstrual pain – known as dysmenorrhea – decreased by 42% in patients who took the medicine and chronic pelvic pain outside of menstruation dropped by 52%. 

Meanwhile, HMI-115 is also in the clinic for androgenic alopecia, known more commonly as pattern hair loss. The $18.5 million series B funding from last year is meant to keep the development of HMI-115 going in the clinic. And now, with the latest FDA go-ahead, it marks a potential shift in endometriosis care, making Hope Medicine one to keep your eyes peeled for. 

ITM Isotope Technologies Munich 

ITM ended 2025 with one of the most advanced late-stage programs in radiopharma. The biotech company’s targeted radiotherapeutic 177Lu-edotreotide (ITM-11) delivered positive phase 3 results, a head-to-head trial against everolimus in patients with inoperable advanced neuroendocrine tumors that have continued to grow despite treatment. The study showed significantly longer progression-free survival and higher response rates with ITM-11, supported by a manageable safety profile. These data formed the basis of a new drug application (NDA) accepted by the FDA in November 2025. 

The coming year will determine whether 177Lu-edotreotide becomes the next approved peptide receptor radionuclide therapy after Novartis’ Lutathera, potentially giving clinicians an additional option for a disease area where systemic treatments remain limited. Approval would also mark an inflection point for ITM, which has been investing in infrastructure beyond a single asset. The company operates a large-scale GMP facility for non-carrier-added lutetium-177 and has established supply agreements for both Lu-177 and actinium-225 with other radiopharma developers, framing it as both a therapeutic developer and a key isotope supplier as the field expands. 

Kailera Therapeutics 

It’s fair to say that, even if it didn’t have a pipeline of late-stage candidates, U.S. biotech company Kailera Therapeutics would be one to watch in 2026 simply because of its incredible $600 million series B financing round in October, providing it with a huge cash runway for research and development.  

Despite the fierce competition, investors have extremely high hopes for the company’s obesity assets, which it licensed from Jiangsu Hengrui Pharmaceuticals last year when it launched with another eye-watering fundraising round of $400 million. The company’s lead candidate, KAI-9531 (in development as HRS9531 in China), is an injectable GLP-1/GIP receptor dual agonist. In July 2025, Kailera and Jiangsu Hengrui Pharmaceuticals reported that the candidate cut average weight by 17.7% versus placebo at 48 weeks across all three dose levels tested in a phase 3 trial in China.  

Additionally, at the start of the year, Kailera and Jiangsu Hengrui Pharmaceuticals posted a more impressive 22.8% mean weight loss from baseline at 36 weeks in a phase 2 study, but this was at a higher 8mg dose than was tested in the phase 3 trial. After its latest raise, Kailera said it would use the funds to launch a global phase 3 clinical program for KAI-9531 by the end of the year, including two trials in adults living with obesity or overweight with comorbidities, with and without type 2 diabetes, and an additional trial in adults living with a body mass index (BMI) of 35 or higher. Kailera previously noted that it would like to investigate how the drug performs at higher doses in global trials to see if its potential can be expanded.  

Kardigan 

After launching in January 2025 with $300 million in series A funding, cardiovascular company Kardigan was back in the headlines in October after bringing in a total of $254 million in series B funding, which turned out to be the third-largest private biotech fundraising of that month.  

The money raised is going toward the company’s three late-stage assets. One of these is called danicamtiv, for which the company shared phase 2b data at the Heart Failure Society of America (HFSA) Annual Scientific Meeting earlier this year. The drug, a cardiac myosin activator licensed from Bristol Myers Squibb and originally discovered by MyoKardia, proved to be well-tolerated with significant improvements in ventricular and atrial function in patients with genetic dilated cardiomyopathy – a condition in which the heart becomes enlarged and cannot pump blood effectively.  

Meanwhile, the company’s other two assets are tonlamarsen, angiotensinogen-targeted bridging therapy in phase 2b studies to interrupt the dangerous cycle of acute severe hypertension, and ataciguat, an oral soluble guanylate cyclase activator in phase 3 studies for the treatment of calcific aortic valve stenosis that could potentially be an alternative to “watchful waiting.”  

According to Kardigan, the new capital from its series B round reinforces its path to multiple data readouts beginning in 2026. 

MaaT Pharma 

MaaT Pharma closed 2025 in a strong position thanks to the phase 3 ARES results for its lead product, MaaT013, in steroid- and ruxolitinib-refractory acute gastrointestinal graft-versus-host disease. The study showed a 62% gastrointestinal response rate and a 54% one-year overall survival, outcomes that are notable in a setting where patients have very few options. These data supported the Marketing Authorization Application submitted to the European Medicines Agency EMA in June 2025, putting MaaT013 on track to potentially become the first approved microbiome-based therapy in Europe.  

The coming year will revolve largely around the regulatory process. An EMA decision is expected around mid-2026, and MaaT has already begun preparing for a potential launch through new commercial partnerships. Beyond the lead asset, the biotech company’s second program, MaaT033, is progressing in a phase 2b trial in patients undergoing stem-cell transplantation, offering another readout to watch as microbiome therapeutics slowly move from concept to clinic. 

If the EMA outcome is positive, MaaT could shift from a late-stage developer to one of the first companies in Europe to commercialize a microbiome therapy, giving the field a clearer sense of its real-world potential. 

MapLight Therapeutics 

After closing its $296.3 million initial public offering (IPO) in October, it seems like the California-based MapLight Therapeutics is gearing up to take on pharma giant Bristol Myers Squibb’s Cobenfy, the only muscarinic agonist approved to treat schizophrenia, deemed a ‘miracle drug.’ 

MapLight’s muscarinic agonist called ML-007C-MA targets a pair of muscarinic receptors, namely, M1 and M4. When they are activated, they regulate neural circuits associated with psychosis and cognition.  

It aims to address positive symptoms such as hallucinations and delusions, negative symptoms – experiencing a lack of motivation and social withdrawal – and cognitive symptoms of schizophrenia without targeting other receptors. As the therapy does not turn on dopamine receptors, which antipsychotic drugs tend to do, it is designed to curtail a flurry of adverse effects like diabetes, weight gain, and even the movement disorder tardive dyskinesia – associated with prolonged antipsychotic use.  

Findings hint that it might be more effective than Cobenfy in improving cognition. iIs dosing is also being tweaked to be more convenient than Cobenfy, a twice-daily pill required to be taken on an empty stomach. 

As 30% of people with schizophrenia do not respond at all to treatments, a drug like ML-007C-MA is hoped to change these statistics for the better. While it may not be first-in-class, ML-007C-MA certainly could topple Cobenfy’s position in the market, and  could be a more effective treatment for people with schizophrenia. 

ML-007C-MA is also being assessed to treat psychosis in people with Alzheimer’s disease, and its other agonist drug ML-004 is intended to improve social interaction and reduce irritability in people with autism spectrum disorder.   

MindMed 

Co-founded by the entrepreneur J.R. Rahn, who suffered from mental health problems and was interested in the Silicon Valley trend of improving focus by microdosing on psychedelic drugs, biotech company MindMed could be one to watch in 2026 in the mental health space. While several other psychedelic drug companies have faced setbacks in recent times, MindMed is smoothly progressing its lead candidate, MM120, through late-stage clinical trials.  

MM120 is an optimized form of the hallucinogenic drug lysergic acid diethylamide (LSD) and acts as a partial agonist at specific serotonin receptors, causing distinctive transient perceptual and emotional effects. By increasing connections between areas of the brain that are not normally connected, the drug enables changes that could treat mental illnesses like depression and anxiety. 

The candidate has received breakthrough therapy designation from the FDA and is currently in phase 3 of development for generalized anxiety disorder and major depressive disorder. In January, MindMed announced that the first patient had been dosed in a second phase 3 trial of MM120 in generalized anxiety disorder, and in April, it dosed the first patient in a phase 3 study of MM120 in major depressive disorder.   

The generalized anxiety disorder studies are expected to produce topline results in the first and second half of 2026, and results of the major depressive disorder study are also expected to come in the second half of next year. 

Novo Nordisk 

After years of riding the GLP-1 boom, 2025 was a reset year for Novo: a profit warning on Wegovy and Ozempic, loss of share to Eli Lilly, a high-profile patent lapse that opens Canada to semaglutide generics from January 2026, and a major restructuring under new CEO Mike Doustdar with 9,000 layoffs and pipeline cuts aimed at saving around 8 billion Danish kroner ($1.3 billion) a year by the end of 2026 and refocusing squarely on obesity, diabetes and a handful of higher-value areas. 

What makes Novo worth watching in 2026 is whether that bet pays off: the company plans to start late-stage trials of amycretin, a dual GLP-1/amylin agonist that has shown up to 22% weight loss with injections and 10 to 14.5% in oral and diabetes trials, alongside strong HbA1c reductions, in both obesity and type 2 diabetes, positioning it as a potential follow-on to semaglutide just as generics emerge. Novo also has several high-impact readouts and potential launches ahead, including the FDA decision on Mim8 for hemophilia A and phase 3 Alzheimer’s results for semaglutide.  

At the same time, Novo is trying to rebuild its edge through big-ticket deals — Akero in MASH for instance. And although Pfizer ultimately won the Metsera bidding war, Novo’s willingness to escalate the contest revealed how determined it is to secure next-generation obesity assets, and how clearly it sees the competitive pressure building around its core franchise. 

moving into 2026, Novo can turn a difficult 2025 into the start of a new growth phase, or the competition and patent pressure can continue to erode its momentum. 

Regenxbio 

The first-ever gene therapy was greenlit by the FDA in 2017, and 46 cell and gene therapies have been cleared since then. Next year could see another gene therapy added to the mix: Regenxbio’s RGX-121. 

The Maryland-based gene therapy developer has designed RGX-121 to address Hunter syndrome, a rare disease characterized by the body’s inability to break down large sugar molecules, which eventually build up in the tissues. This is due to the lack of the iduronate-2-sulfatase enzyme produced by a gene present in the central nervous system. RGX-121 contains the gene delivered via an adeno-associated virus (AAV) vector. 

Encouraging 12-month data showed that the one-time treatment reduced the levels of a sugar called heparan sulfate in cerebrospinal fluid – a fluid that cushions the brain and spinal cord – by 80%, hitting the primary endpoint. 

Meanwhile, its other gene therapy RGX-202 could treat Duchenne muscular dystrophy, a rare genetic disorder that leads to muscle loss and weakness. To add to that, AbbVie-partnered ABBV-RGX-314 is in the clinic to treat wet age-related macular degeneration (AMD), an eye condition that causes blurred vision. 

To fund this pipeline, the American biotech raked in $150 million in a royalty deal in May meant to carry the company through to 2027. 

Having filed for RGX-121’s approval with the FDA in 2024, the regulators will make their decision in February 2026. As the Hunter syndrome community and the gene therapy space wait with bated breath, if approved, Regenxbio’s medicine would be the first of its kind to receive the FDA nod to treat the rare inherited disorder.  

Reunion Neuroscience 

Perinatal depression occurs during pregnancy and after childbirth, and around one in seven people are affected by the mood disorder. While serotonin reuptake inhibitors (SSRIs) are considered the first line of treatment, they are associated with limited efficacy and harmful side effects, and they require continuous dosing. 

Ontario-based Reunion Neuroscience is developing a psilocybin-like molecule to treat postpartum depression. Psilocybin is a naturally occurring psychedelic found in more than 200 mushrooms. However, unlike psilocybin, RE104 is designed to deliver a shorter psychedelic experience.  

Reunion’s candidate RE104 is a prodrug – a drug that needs to be activated, usually by an enzyme – of 4-OH-DiPT. It switches on a specific receptor, which acts as a key target for psychedelic medicines. In a phase 2 study, a 30 mg dose of the therapy had a 23-point reduction from the baseline after a week, according to the Montgomery-Asberg Depression Rating Scale (MADRS) – a measure of the severity of depressive symptoms. 

Also, early data from a lactation study found less than 0.1% of the 30 mg RE104 present in mothers’ breastmilk, which is below the level that might potentially cause risk to the infant. This data is, of course, subject to FDA review.  

Meanwhile, RE104 is being evaluated to potentially treat adjustment disorder in cancer and generalized anxiety disorder. Adjustment disorders are often linked to a stressful event, and many people with cancer experience it due to the ongoing stressors associated with diagnosis and treatment.  

The Canadian biotech company secured $133 million in series A financing in September, which will fuel phase 3 studies of RE104 for postpartum depression in 2026. Whether it is a true contender against SSRIs, we will have to see how things wrap up in the clinic.