Startup is a term we hear often as more and more people offer their exclusive solutions to issues and problems. But does anyone think about what is behind Startup and its promotion to the masses? Probably not, but it’s worth considering. After all, behind it is not just a year, but decades of careful and meticulous work.
Not all starts are successful, because after you create it, a new phase begins: presentation and recognition. Explaining yourself and telling other companies why your startup deserves investment and why it’s important is sometimes even harder than developing it, because in a moment everything can just fall apart.
In the 21st century, a country’s ability to develop new science and turn it into innovative technology, to establish itself and turn it into a successful, sustainable, income-generating business is paramount to a country’s development and well-being. In this day and age where new diseases and viruses are flooding the world, everyone is waiting for salvation and for the person who will find the solution. Everything is evolving rapidly and biotech innovations are gaining pace and surprising the world with extraordinary approaches to complex problems.
Best Biotech Startups
Longevity & Cellular Reprogramming
NewLimit: The “Software” Approach to Aging
NewLimit is based on the idea that aging is not just “wear and tear,” but a software glitch. Every cell in your body has the same DNA (the hardware), but its “epigenetic markers” (the software) tell it whether to act like a young skin cell or an old, failing liver cell.
They use mRNA-delivered transcription factors to “reprogram” the epigenome. Unlike classic stem cell research that turns a cell back to a “blank slate” (which can cause cancer), NewLimit aims for partial reprogramming—making an old liver cell function like a young liver cell without losing its identity.
2025 Focus: They are currently targeting three specific areas:
- Liver: Restoring the ability of aged hepatocytes to process fats and alcohol.
- Immune System: Rejuvenating T cells to better fight off infections and cancer in the elderly.
- Vascular: Targeting “Endothelial” cells (blood vessel lining) to treat chronic kidney disease.
The “Lab-in-a-Loop”: They use a massive AI feedback loop where machine learning predicts which genetic “switches” to flip, robots test them in the lab, and the results are fed back into the AI to refine the next round of experiments.
BioAge Labs: The “Data-First” Approach
While others start with a theory, BioAge starts with human data. They have exclusive access to biobanks containing blood samples and health records of thousands of people tracked over 45+ years.
They use AI to look at “super-agers”—people who lived to 90+ with high physical and cognitive function—and identify the specific molecules in their blood that kept them young.
In 2025, their primary focus is an oral drug that inhibits NLRP3, a protein responsible for “inflammaging” (chronic age-related inflammation).
2025 Milestone: They recently released positive Phase 1 clinical data showing the drug is safe and effective at reducing inflammatory markers in patients with obesity and cardiovascular risk.
Secondary Pipeline: They are developing APJ agonists—drugs that mimic the beneficial effects of exercise on the heart and muscles, specifically to prevent “sarcopenia” (muscle wasting) in the elderly.
Loyal (Cellular Longevity): The “Real-World” Approach
Loyal’s strategy is the most pragmatic: if you want to prove a longevity drug works in humans, it might take 50 years to see the results. If you prove it in dogs, you can see results in 3–5 years.
Dogs age similarly to humans and share our environment. By getting the first-ever FDA approval for a longevity drug in dogs, they are building the regulatory “playbook” for human longevity drugs.
Current Pipeline:
- LOY-001 (Injection): For large-breed dogs (like Great Danes) who die young. It targets the IGF-1 growth hormone, which is overexpressed in big dogs and linked to shorter lifespans.
- LOY-002 (Daily Pill): For senior dogs of all sizes. It is a “metabolic modulator” that mimics the benefits of a low-calorie diet to improve energy and healthspan.
The Human Pivot: In 2025, Loyal is using the “gold mine” of data from their STAY study (the largest clinical trial in animal health history) to begin selecting human drug candidates. The idea is that the metabolic pathways they are fixing in dogs are nearly identical to those in humans.
Next-Gen Gene & Cell Therapy
Tune Therapeutics: The “Epi-Editor”
Tune is pioneering the shift from “cutting” DNA to “tuning” it. Their TEMPO platform doesn’t change the underlying genetic code; instead, it uses the body’s natural “volume knobs” (epigenetic markers).
In early 2025, Tune secured $175M and named veteran drug hunter John McHutchison as CEO. They are now moving into the clinic with TUNE-401, which has received regulatory approval for trials in New Zealand and Hong Kong.
Traditional treatments for HBV rarely “cure” the disease because the virus leaves behind a stable “reservoir” (cccDNA) in the liver. Tune’s therapy uses a lipid nanoparticle (LNP) to deliver an epi-silencer that permanently “turns off” this viral DNA, aiming for a functional cure with a single dose.
Why it matters: Because it doesn’t break the DNA strand, there is a significantly lower risk of “off-target” mutations compared to CRISPR.
SpliceBio: The “Protein Zipper”
AAV (the standard delivery vehicle for gene therapy) has a “small trunk”—it can’t carry genes larger than ~4.7kb. This leaves many diseases, like Stargardt (blindness), untreatable.
SpliceBio uses Engineered Inteins. They split a large gene into two smaller pieces, pack them into two separate AAVs, and send them into the cell. Once inside, the two halves “zip” together at the protein level to form the full, functional gene.
They recently raised a $135M Series B led by Sanofi and EQT. More importantly, they dosed the first patient in their Phase 1/2 ASTRA study for Stargardt Disease (targeting the massive ABCA4 gene).
With the new funding, they are moving beyond eyes into neurology, where many critical genes (like those for certain types of Muscular Dystrophy) are too large for standard delivery.
Be Biopharma: The “B Cell” Factory
While CAR-T therapy uses T cells to kill, Be Biopharma uses B cells to build. B cells are the body’s natural protein factories (usually making antibodies). Be “re-wires” them to produce therapeutic proteins instead.
In early 2025, they closed a $130M Series C and transitioned into a multi-program clinical stage company. They recently dosed the first participant in the BeCoMe-9 trial for Hemophilia B. The goal is for the patient’s own B cells to continuously produce “Factor IX,” potentially eliminating the need for regular injections.
Upcoming Pipeline: They are now testing BE-102 for Hypophosphatasia (a rare bone disease) and investigating using B cells to deliver “anti-tumor biologics” directly to cancer sites, which could be much safer than systemic chemotherapy.
AAVantgarde Bio: The “Twin-Vector” Specialists
Based in Italy, this startup is the primary competitor to SpliceBio. They are also tackling the “large gene” problem but focusing specifically on the hardest-to-treat inherited retinal diseases (IRDs).
They closed a massive $141M Series B in late 2025 to scale their two lead clinical programs.
The Dual-Platform Approach: They use two distinct platforms:
- AAVB-081: Targets Usher Syndrome Type 1B (which causes both deafness and blindness). Their LUCE Phase 1/2 trial is currently reporting “early efficacy signals” and a strong safety profile.
- AAVB-039: Their program for Stargardt Disease, providing a direct “head-to-head” race with SpliceBio.
AAVantgarde’s “hybrid” vectors are designed to maximize the efficiency of how the two halves of a gene recombine, which is often the bottleneck in dual-vector therapy.
AI & Software-Driven Biotech
Insilico Medicine: The AI IPO Pioneer
Insilico is the “gold standard” for proving that AI can actually produce drugs that work in humans. On December 30, 2025, Insilico successfully listed on the Hong Kong Stock Exchange (HKEX), marking the largest biotech IPO of the year in Hong Kong with a market cap of approximately $2.5 billion.
Their lead candidate, Rentosertib (ISM001-055), became the first AI-designed drug to complete a Phase 2a clinical trial (for Idiopathic Pulmonary Fibrosis). Results published in Nature Medicine in 2025 showed significant gains in lung function, providing the first “clinical proof-of-concept” for the entire AI drug discovery field.
Pharmaceutical Superintelligence: In late 2025, they launched Life Star 2, a fully autonomous robotic lab that uses AI to not only design drugs but also synthesize and test them 24/7 without human intervention.
Benchling: The Central Nervous System of Biotech
Benchling is the software platform that almost every startup on this list uses to organize their R&D. If Big Pharma is a factory, Benchling is the digital blueprint.
As of 2025, over 60% of the world’s top 50 biopharma companies and thousands of startups use Benchling. It has effectively replaced paper notebooks and fragmented spreadsheets with a unified cloud-based data layer.
Benchling recently launched “Benchling Connect AI,” which automatically pulls data from lab instruments (like DNA sequencers and liquid handlers) and uses LLMs to draft experiment summaries and flag anomalies in real-time.
By centralizing R&D data, Benchling allows companies to train their own proprietary AI models, making it the essential infrastructure for the “AI-native” biotech era.
Kailera Therapeutics: The GLP-1 “Challenger”
Kailera emerged from stealth in 2024 and became a powerhouse in 2025, aiming to take on giants like Eli Lilly and Novo Nordisk in the weight-loss market.
$600M Series B: In October 2025, Kailera closed one of the largest private funding rounds of the year to advance its obesity pipeline.
The “Best-in-Class” Injectable: Their lead asset, KAI-9531, is a dual GLP-1/GIP receptor agonist. In Phase 2 trials, it showed a 22.8% mean weight loss over 36 weeks—outperforming the 20.9% seen in early trials of Eli Lilly’s Zepbound.
The company is initiating Global Phase 3 trials by the end of 2025. They are also developing an oral version of the drug and a “tri-agonist” (GLP-1/GIP/Glucagon) to maximize fat burning while preserving muscle.
Vivodyne: The “Animal Testing” Disruptor
Vivodyne is solving the “90% failure rate” in drug development by moving from testing on mice to testing on lab-grown human organs.
Using a $77M Series A led by Khosla Ventures in February 2025, Vivodyne has scaled its platform to grow over 100,000 functional human tissue samples at a time. These are not simple cell clusters, but complex, vascularized organ models (liver, lung, bone marrow) that can actually “breathe” and circulate nutrients.
Their “Data Engine” robots dose these tissues with drugs and use high-speed imaging to capture how the human immune system responds. This generates more human-specific data in a month than decades of animal studies.
San Francisco Expansion: In mid-2025, they opened a massive 23,000-square-foot robotic facility in San Francisco to partner with Big Pharma companies looking to bypass animal models following the FDA’s Modernization Act 2.0.
Oncology & Precision Medicine
ARTBIO: The Alpha-Emitter Revolution
While traditional radiation (Beta therapy) acts like a “carpet bomb,” ARTBIO uses Alpha particles, which are like “guided missiles” with a very short range but immense destructive power.
Lead Program (AB001): In late 2025, ARTBIO dosed the first patient in their ARTISAN Phase 1 trial for metastatic prostate cancer. Unlike the currently approved Pluvicto, which uses Lutetium-177 (Beta), AB001 uses Lead-212 ($Pb^{212}$).
Alpha particles deliver a much higher “linear energy transfer,” causing irreversible double-stranded DNA breaks in cancer cells while sparing the healthy tissue just millimeters away.
Radiopharmaceuticals usually have a very short shelf-life. ARTBIO’s AlphaDirect™ technology allows them to produce $Pb^{212}$ on-site at hospitals, solving the logistics “nightmare” that has plagued this sector.
Tubulis: The “Smart Bomb” Engineering
Antibody-Drug Conjugates (ADCs) are often called “biological missiles.” Tubulis is solving the biggest problem with ADCs: they often fall apart in the blood, causing toxic side effects before they reach the tumor.
Tubulis uses a unique “cysteine-selective” conjugation method. This ensures the toxic payload stays glued to the antibody until it is inside the cancer cell.
At the ESMO 2025 Congress, Tubulis presented data for TUB-040, showing a massive 59% overall response rate in patients with platinum-resistant ovarian cancer.
Beyond their own pipeline, their $361M Series C is fueling a major partnership with Bristol Myers Squibb, using Tubulis’ technology to build BMS’s next generation of cancer drugs.
OnCusp Therapeutics: The “Bridge” Builder
OnCusp doesn’t just discover drugs; they “rescue” them. They identify high-potential assets in early-stage labs (often in China or academia) and use their elite clinical team to navigate the “Valley of Death”—the difficult jump from the lab to human trials.
In June 2025, OnCusp announced encouraging results from their Phase 1a trial for CUSP06, an ADC targeting CDH6. This protein is overexpressed in ovarian and kidney cancers.
In February 2025, the FDA granted Fast Track Designation to CUSP06. Early data showed it was effective even in patients who had failed other modern therapies, including Elahere.
By acting as a specialized “translational” engine, OnCusp can bring drugs to market 2–3 years faster than traditional biotech models.
Mirador Therapeutics: Precision Immunology
Mirador is the new “supergroup” of biotech, founded by the team that sold Prometheus Biosciences to Merck for $10.8 billion. They are applying the “Prometheus Playbook” to the entire field of immunology.
Mirador360™ Engine: This platform uses human genetics and machine learning to identify which specific “pathways” (like IL-23 or TL1A) are driving a patient’s inflammation. Instead of guessing which drug will work, they use a “companion diagnostic” to test the patient first.
In August 2025, they initiated a Phase 2 study for MT-501, a drug for Ulcerative Colitis and Crohn’s Disease.
They have secured exclusive licenses to massive genetic databases (including 23andMe data) to find the “hidden” genetic drivers of immune diseases that other companies miss.
Specialized Platforms & Diagnostics
Mammoth Biosciences: The CRISPR “Swiss Army Knife”
Founded by Jennifer Doudna, Mammoth is expanding CRISPR’s utility in two directions: making it small enough to fit in any delivery vehicle and fast enough to diagnose diseases in minutes.
In 2025, Mammoth presented data at ASGCT showing that their NanoCas system—the first efficient “ultra-compact” gene editor—can be delivered via a single AAV vector to edit muscle tissue. Because it is so small, it overcomes the “size limit” that has hindered CRISPR treatments for Duchenne Muscular Dystrophy (DMD).
Their diagnostic platform uses CRISPR as a molecular shredder. When a specific viral or bacterial sequence is found, the Cas enzyme “snips” a reporter molecule, creating a signal. In late 2025, they revealed programs for real-time biothreat detection and point-of-care testing for respiratory infections.
They are currently in a multi-year collaboration with Regeneron to develop in vivo gene silencing for liver and muscle diseases.
Freenome: The AI Multi-Cancer Detectors
Freenome is a leader in the “liquid biopsy” race, using a blood draw to catch cancer at Stage 1, when it is most curable.
The Roche & Exact Sciences Deals: 2025 was a massive year for Freenome’s commercialization. They signed an $885M deal with Exact Sciences (the makers of Cologuard) for U.S. rights to their colorectal cancer (CRC) test, and a separate strategic alliance with Roche to launch their “kitted” tests internationally.
PREEMPT CRC Success: Their pivotal study of 49,000 adults showed an 81.1% sensitivity for colorectal cancer. They have submitted their final modules to the FDA, with a full commercial launch anticipated for 2026.
Unlike competitors who only look at DNA, Freenome’s AI analyzes multiomics—DNA, proteins, and metabolic markers—to get a more complete picture of the “tumor microenvironment.”
BillionToOne: The “Molecular Counter”
BillionToOne reached a $4.97B market cap in late 2025 following a successful IPO. Their secret sauce is their QCT (Quantitative Counting Template) platform, which can count DNA molecules with single-molecule precision.
This is the only non-invasive prenatal test (NIPT) that can screen for single-gene disorders (like Cystic Fibrosis) from the mother’s blood without needing a sample from the father. In 2025, they expanded this to cover 14 recessive conditions, capturing 15% of the total U.S. prenatal market.
They are now aggressively expanding into cancer. Their Northstar Response test allows oncologists to see if a treatment is working by “counting” the drop in circulating tumor DNA (ctDNA) significantly faster than a traditional CT scan could.
The company reported nearly 100% year-over-year revenue growth, projecting $295M+ for the full year of 2025.
Hera Biotech: Solving the “Silent Epidemic”
Endometriosis affects 1 in 10 women, but it currently takes an average of 8 years to diagnose because it requires invasive surgery. Hera is ending that delay.
Hera has developed a molecular diagnostic that uses a simple tissue sample (collected during a regular OB-GYN visit) to identify the gene expression “signature” of endometriosis.
In late 2024/early 2025, Hera acquired the AI assets of Scailyte, combining the world’s two largest single-cell datasets for endometriosis.
They are currently using their Series A funds to launch their first product into the U.S. fertility market, aiming to help women struggling with infertility find answers without undergoing laparoscopy.
Diagonal Therapeutics: The “Agonist” Pioneers
Most drugs work by “blocking” something (antagonists). Diagonal is doing the opposite: they make antibodies that turn signals back on.
Their lead candidate targets Hereditary Hemorrhagic Telangiectasia (HHT), a rare disease where blood vessels form incorrectly and rupture. Their agonist antibody “zips” two receptors together to restore the signaling pathway that keeps blood vessels stable.
In mid-2025, DIAG723 received FDA Orphan Drug Designation. Preclinical data presented at the EHA Congress showed the drug could not only prevent but actually reverse existing vascular malformations.
Their computational engine allows them to sift through billions of antibody configurations to find the rare few that act as “keys” to unlock specific cellular functions.
Synthetic Biology & Sustainability
Birch Biosciences: The “Infinite” Plastic Recycler
Birch is moving past the limitations of mechanical recycling (which usually degrades plastic until it’s eventually thrown away) by using engineered enzymes that treat plastic as a raw material rather than waste.
Birch uses generative AI to design enzymes that can “unzip” complex plastic polymers like PET (bottles) and Polyurethane (foam) back into their original chemical building blocks (monomers).
In June 2025, Birch entered a global patent license agreement with NREL (National Renewable Energy Laboratory) to commercialize a breakthrough enzymatic deconstruction technology. This allows them to recover high-value monomers that are chemically identical to “virgin” plastic made from oil.
Unlike traditional recycling, which involves melting plastic at high temperatures (emitting CO2 and weakening the material), Birch’s process runs at low temperatures and reduces carbon emissions by over 70%. This enables a “circular” model where a plastic bottle can be recycled back into the same high-quality bottle indefinitely.
They recently secured a $1M NSF SBIR Phase II Award and are working with DARPA to adapt their AI-driven enzyme platform for a wider range of military and industrial plastics.
C16 Biosciences: The “Palmless” Revolution
Palm oil is in 50% of supermarket products, but its production is the leading cause of tropical deforestation. C16 Biosciences uses precision fermentation to “brew” a sustainable alternative that requires zero tropical land.
Their lead product, Torula oil, is produced by feeding a specific strain of yeast sugar in large fermentation tanks. The result is an oil that is chemically and functionally almost identical to palm oil.
After successfully launching in the beauty sector (where their oil is used in high-end skincare to repair skin barriers), C16 is now moving into food-grade fats. In late 2024 and throughout 2025, they received $4.5M in fresh funding, including a major grant from the Bill & Melinda Gates Foundation.
In 2025, they signed a Letter of Intent with ScaleUp Bio in Singapore to utilize their food-grade fermentation facilities. This moves C16 closer to replacing palm oil in global staples like chocolate, bakery items, and infant formula.
By using non-agricultural feedstocks (like food waste or industrial byproducts) to feed their yeast, C16 is decoupling oil production from the need for arable land, offering a “deforestation-free” supply chain for Big Food.
Conclusion: biotech startups
These startups are among the many discoveries that are taking biotechnology to a new level and giving people even more chances to be healthy. Looking at this technological development, we want to believe that in the near future we will be able to fight dangerous viruses and diseases quickly.
Now, it is also worth mentioning that the startup is spreading to the masses. This is quite difficult because every day more new breakthroughs turn tired norms upside down. So what to do in this whirlwind of information? The best way is to make yourself known and not be afraid of it. PR is a key point in this process, so if you are looking for a way to tell the world about yourself and share your amazing projects that can change people’s lives, don’t hesitate to contact our company who will be happy to help!
