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The BIO International Convention 2025, held in Boston this June, once again underscored the region’s leading role in the global biotech landscape. As thousands of industry leaders gathered to exchange ideas and explore new partnerships. But after the convention wraps up, biotech innovation continues to thrive in Boston. That’s why today, we’re taking a closer look at 14 biotech companies shaping the future from this powerhouse hub. The Boston region has sprouted many influential public biotech companies. One of the most successful in recent years was Moderna, the giant messenger RNA (mRNA) specialist incubated by the venture capital (VC) firm Flagship Pioneering. Moderna would go on to produce one of the first COVID-19 vaccines in 2020. Another prestigious name, Vertex Pharmaceuticals, one of the biotechs with a market capitalization above $100 billion, is also headquartered in Boston. Without further ado, here are 14 companies to keep an eye on in 2025 and beyond. Akouos Founded: 2016 Focus area: Gene therapy for inner-ear disorders Lead candidate: AK-OTOF (gene therapy for otoferlin-related hearing loss) Akouos is a Boston-based biotech company developing gene therapies for sensorineural hearing loss, a condition with no approved genetic treatments yet. Akouos built its pipeline on a proprietary platform that uses adeno-associated viral (AAV) vectors, specifically engineered for inner-ear delivery. Its lead candidate, AK-OTOF, is an AAV-based gene therapy designed to restore otoferlin, a protein critical for the synaptic transmission of sound in the auditory system. The therapy targets individuals with mutations in the OTOF gene, which can lead to congenital deafness. Akouos initiated a phase 1/2 trial for AK-OTOF in 2022, and in January 2024, the company reported a striking early result: a deaf 11-year-old boy who received the therapy regained normal levels of hearing within a month of treatment. This result is one of the most dramatic clinical responses observed so far in the field of gene therapy. Big pharma didn’t wait for these positive results to show interest in Akouos. Indeed, the Boston-based biotech was acquired by Eli Lilly in late 2022 for $487 million upfront, with additional milestone-based payments bringing the deal value up to $610 million. Beyond AK-OTOF, the company is advancing several preclinical programs, including candidates for Usher syndrome, GJB2-related hearing loss, and vestibular schwannoma. Alterome Therapeutics Founded: 2022 Focus area: Precision small-molecule oncology Lead candidates: ALTA‑2618 and ALTA‑3263 Alterome Therapeutics is a Boston-area biotech founded in 2022, using its Kraken platform, a structure-guided, machine learning system, to design small molecules that selectively target mutated forms of cancer proteins. This allows the company to develop compounds that differentiate cancer-specific mutations from the normal versions found in healthy cells, potentially improving efficacy while reducing side effects. Their pipeline includes two clinical-stage assets: ALTA‑2618, a mutation-selective inhibitor for AKT1 E17K-driven cancers, is currently in a phase 1/1b study enrolling patients with advanced solid tumors. ALTA‑3263, an oral pan-KRAS inhibitor targeting over 90% of KRAS mutations, entered phase 1/1b dosing in early 2025. In April 2024, Alterome closed a $132 million series B, led by Goldman Sachs Alternatives, bringing its total funding to nearly $232 million. The company stated that these proceeds would support advancing its two lead programs into the clinic within 12 months; it has since delivered on that goal. Ascidian Therapeutics Founded: 2020 Focus area: RNA exon-editing therapeutics Lead candidate: ACDN‑01 Ascidian focuses on RNA exon editing, a novel therapeutic approach that “rewrites” segments of RNA before they're translated into protein, ideal for diseases caused by mutations in large genes that are hard to edit at the DNA level. Its lead candidate, ACDN‑01, is the first-ever clinical-stage RNA exon editor, targeting Stargardt disease, an inherited retinal degeneration caused by ABCA4 gene mutations. In January 2024, the U.S. Food and Drug Administration (FDA) cleared its investigational new drug (IND), granting both fast track and rare pediatric disease designations, and the company is now enrolling patients in the phase 1/2 STELLAR trial for subretinal delivery. The company attracted big pharma players, too. In June 2024, Roche signed a research and license agreement with Ascidian for neurological diseases, paying $42 million upfront with up to $1.8 billion in milestones. Under the deal, Ascidian runs discovery and preclinical work, while Roche handles clinical progression and commercialization. Cellarity Founded: 2017 Focus area: Small molecules that reset disease-associated cellular behaviors Lead candidate: CLT‑1081 Cellarity was spun out of Flagship Pioneering with a fresh take: treat disease by reprogramming cell-state behaviors rather than targeting single proteins. Their platform integrates high-resolution single-cell data and proprietary computational models, called “Cellarity maps,” to uncover and reverse the cellular signatures of disease The company’s most advanced candidate is an oral small molecule designed to boost fetal hemoglobin in sickle cell disease by shifting red blood cell precursors toward healthy development. The program is still preclinical but should soon enter the clinic. Beyond sickle cell, their pipeline also includes discovery-stage programs targeting myelofibrosis, immuno-oncology, and metabolic dysfunction-associated steatohepatitis (MASH). In January 2024, it expanded a strategic collaboration with Novo Nordisk to apply their platform to MASH, a chronic liver disease, with the partnership potentially providing up to $532 million. Cellarity has raised over $270 million in venture funding, including a $121 million series C in 2022. While still early in terms of clinical development, the strong funding and the Novo deal make Cellarity a biotech to watch closely. Delix Therapeutics Founded: 2019 Focus area: Non-hallucinogenic neuroplastogens Lead candidate: DLX‑001 Delix is translating neuroscience into small-molecule drugs that promote brain plasticity without the hallucinogenic effects of classic psychedelics. The Boston biotech’s lead candidate is DLX‑001, a next-gen psychoplastogen inspired by compounds like dimethyltryptamine and ketamine, but modified to avoid dissociation and hallucinations. DLX‑001 began phase 1 studies in mid-2023, and results have shown that DLX‑001 successfully penetrates the brain, is well-tolerated, and exhibits dose-dependent pharmacodynamic signals, with no hallucinogenic or dissociative side effects reported. Encouraged by the data, Delix launched a phase 1b trial in patients with major depressive disorder in 2024. The company also said it planned a phase 2 study in mid-2025. Delix is rapidly building its pipeline beyond DLX‑001: a second clinical candidate, DLX‑159, showed robust preclinical plasticity effects and is heading into IND-enabling studies. Plus, the company secured an $825,000 Department of Defense grant to evaluate neuroplastogen treatments for hearing loss. ElevateBio Founded: 2017 Focus area: In vivo allele-selective gene editing for monogenic brain disorders Lead candidate: LETI‑101 The biotech company ElevateBio was founded in 2017 in Waltham, a city close to Boston. The gene and cell therapy developer has consistently bagged outsized funding rounds, topped by an impressive $525 million series C round in March 2021. It was only the beginning for ElevateBio, as the company raised $401 million in its series D financing round in 2023. It is focused on enabling others to build and scale cell and gene therapies, rather than advancing its own clinical pipeline. Built around its BaseCamp facilities and its Life Edit gene-editing unit, the company integrates gene editing, vector production, RNA and protein engineering, next-generation sequencing, and cGMP manufacturing, an all-in-one ecosystem attracting partners such as Moderna or Novo Nordisk. Beyond this partnership model, the Boston biotech company shared compelling preclinical data on LETI-101 in February. LETI‑101 is a Huntington’s disease candidate developed by its Life Edit division. Delivered via lipid nanoparticles in rodents, LETI‑101 demonstrated robust brain editing with low immunogenicity, suggesting strong in vivo delivery potential. The company did specify that these results “support advancement into clinical development through ElevateBio’s partnership business model,” so it doesn’t look like it will be advancing the candidate on its own. ElevateBio is also making moves to improve its capabilities. In March 2025, ElevateBio entered a multi-year collaboration with Amazon Web Services (AWS) to use its cloud infrastructure and SageMaker tool to train protein language models on Elevate’s proprietary CRISPR platform. Incendia Therapeutics Founded: 2015 (formerly Parthenon Therapeutics) Focus area: Tumor microenvironment modulation for solid tumors Lead candidate: PRTH‑101 Incendia Therapeutics is developing antibody-based therapies that reprogram the tumor microenvironment to improve the immune system’s ability to fight cancer. Its lead candidate, PRTH‑101, is a monoclonal antibody that targets DDR1, a collagen-binding receptor overexpressed in many tumors. PRTH‑101 disrupts DDR1’s role in aligning and organizing collagen fibers around the tumor, structures that often prevent immune cells from infiltrating. This remodeling effect is designed to convert immune-excluded tumors into ones more likely to respond to immunotherapy. The candidate began clinical development in 2023 with a phase 1a/1b trial in advanced solid tumors, both as a monotherapy and in combination with pembrolizumab. The study demonstrated a favorable safety profile and early signals of activity. In 2024, the company launched a phase 1c trial, now enrolling patients to further evaluate PRTH‑101 across a range of solid tumors and dosing strategies. Incendia is among the biotechs aiming to solve one of immuno-oncology’s persistent challenges: how to make the tumor microenvironment more permissive to treatment. Life Biosciences Founded: 2017 Focus area: Age-related disease therapies Lead candidate: ER‑100 Life Biosciences, founded in Cambridge, is developing ER‑100, an AAV-based gene therapy designed to restore vision by reprogramming aged nerve cells in the eye. The therapy is built on the concept of partial cellular reprogramming, using a set of transcription factors – Oct4, Sox2, and Klf4, also known as Yamanaka factors – which can reset gene expression patterns in cells without erasing their identity. This approach aims to reverse cellular aging and repair damaged tissue while preserving cell function. In October 2024, at the American Academy of Ophthalmology Annual Meeting, the company presented data showing that ER‑100 significantly restored visual function and increased axon density in a non-human primate model of optic nerve injury, reinforcing earlier findings from earlier studies. Financially, Life Biosciences completed a series C round of $82 million in January 2022, bringing its total funding to around $158 million at that time. The company is currently on track to begin first-in-human trials in the second half of 2025. The next chapter for this Boston-area biotech will be about turning preclinical promise into clinical progress. MOMA Therapeutics Founded: 2020 Focus area: Small-molecule inhibitors targeting DNA-repair Lead candidate: MOMA‑313 MOMA Therapeutics applies its proprietary KNOMATIC platform to drug-challenging targets known as molecular machines, proteins like helicases and polymerases that change shape during their function. These proteins are deeply implicated in DNA repair and cancer survival but have historically been very difficult to target with drugs. Their lead candidate, MOMA‑313, is a highly selective oral inhibitor of DNA polymerase theta, an enzyme playing a key role in an alternative DNA-repair pathway. This drug is being developed both on its own and alongside a PARP inhibitor. The goal is to exploit a concept called synthetic lethality, where blocking two different DNA repair pathways at once causes cancer cells to die, while healthy cells remain less affected. The first patient was dosed in August 2024 in a phase 1 study, and early safety and dose optimization data are expected by mid-2026. MOMA is also developing a second drug, MOMA‑341, which targets an enzyme called Werner helicase. This enzyme plays a role in how cells repair DNA, and the drug is designed to treat cancers with a specific genetic trait known as microsatellite instability, often found in certain colorectal and endometrial tumors. The company is preparing for IND submission in early 2025. The company also in-licensed MOMA‑989, a next-gen PARP1 inhibitor also anticipated to enter IND phase by late 2025. Neumora Therapeutics Founded: 2019 Focus area: Precision neuroscience for neuropsychiatric and neurodegenerative diseases Lead candidate: Navacaprant (NMRA‑140) This biotech company is based just outside of Boston, in Watertown. It is developing targeted therapies for brain disorders using a data-driven approach that integrates genetics, brain imaging, and digital biomarkers. Its lead candidate, navacaprant, is designed to treat major depressive disorder (MDD) by modulating the kappa-opioid receptor, a pathway involved in mood regulation and emotional resilience. In January 2025, navacaprant unfortunately failed to meet primary or key secondary endpoints in its first pivotal trial (KOASTAL‑1), triggering a steep drop in the company’s stock. However, depression trials are complex and often subject to high placebo responses, which can mask treatment effects. Neumora is continuing two additional phase 3 studies, KOASTAL‑2 and KOASTAL‑3, with refinements to trial design and additional patient monitoring. Results are expected in the first half of 2026, and could still validate the drug's potential. While these developments do represent a significant setback for the company, let’s not bury the treatment yet and wait and see if the adjustments will make a difference. Meanwhile, Neumora is progressing other programs in its pipeline. NMRA‑511, a vasopressin 1a receptor antagonist, is in a phase 1 trial for agitation in Alzheimer’s disease, with results expected in late 2025. The company is also advancing preclinical assets, including a second-generation M4 receptor modulator for schizophrenia and an NLRP3 inflammasome inhibitor targeting neuroinflammation in Parkinson’s disease. Orna Therapeutics Founded: 2019 Focus area: Circular RNA therapies Lead candidate: In vivo CAR-T Orna Therapeutics is developing therapies using circular RNA, an alternative to conventional linear mRNA. Circular RNAs offer an advantage: they are inherently more stable, which can translate into longer-lasting protein expression in cells. Orna’s proprietary platform, FoRCE, enables the design and production of these molecules along with their delivery via lipid nanoparticles. Its lead program is an in vivo CAR-T therapy that aims to reprogram a patient’s immune cells directly inside the body, removing the need for cell extraction and manufacturing steps. Instead of delivering the CAR protein or modified cells, Orna delivers circular RNA that instructs the patient’s own T cells to produce the CAR receptor. At the PEGS and ASGCT conferences in 2023, Orna presented preclinical data demonstrating that its CAR-T therapy could eradicate tumors in mice at doses 10 to 20 times lower than earlier versions. More recently, at ASGCT in May 2025, its in vivo CAR-T approach showed strong B-cell depletion in mice and non-human primates at low doses. The company has continued to expand its pipeline and capabilities. In May 2024, it acquired ReNAgade Therapeutics, bringing in additional delivery technology. It also maintains a collaboration with Merck to co-develop vaccines and RNA-based therapeutics. Orna expects to enter the clinic in 2026 with a CD19-targeting candidate in autoimmune disease, followed by a BCMA program in oncology. Remix Therapeutics Founded: 2019 Focus area: Small molecules that modulate RNA processing Lead candidate: REM‑422 Remix Therapeutics is developing small-molecule drugs that modulate how RNA is processed inside cells. Its proprietary platform allows the company to design compounds that can alter splicing, degrade disease-driving transcripts, or introduce disruptive elements into RNA. Its lead candidate, REM‑422, is an oral small molecule designed to degrade the RNA encoding MYB, a transcription factor involved in cancers such as acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), and adenoid cystic carcinoma (ACC). In 2024, the company began two phase 1 trials, one for AML/MDS, the other for ACC, after receiving orphan drug designations for both indications from the FDA. Remix has raised over $200 million in funding to date, including an $81 million series A in 2021 and a $70 million Series B in 2022. More recently, the company closed a $60 million funding round. Seaport Therapeutics Founded: 2024 Focus area: Oral neurosteroid and neuroplasticity therapies Lead candidate: SPT‑300 Seaport Therapeutics launched in April 2024 with a $100 million series A, spun out from PureTech to develop neuropsychiatric drugs enabled by its proprietary Glyph platform, which enhances oral bioavailability by routing compounds through the lymphatic system. Its lead candidate, SPT‑300, is an oral prodrug of allopregnanolone designed to enable daily dosing for mood and anxiety disorders. In phase 1, SPT‑300 demonstrated favorable safety, pharmacokinetics, and evidence of GABA_A receptor engagement across multiple dosing regimens. A phase 2a trial in healthy volunteers using the Trier Social Stress Test showed a significant reduction in cortisol levels compared to placebo. Following these results, Seaport closed an oversubscribed $225 million series B round in October 2024, bringing total financing to $325 million. Seaport’s pipeline also includes: SPT‑320, a prodrug of agomelatine designed to reduce dose-related liver issues in generalized anxiety disorder. SPT‑348, a non-hallucinogenic neuroplastogen prodrug aimed at mood and neuropsychiatric disorders. Tessera Therapeutics Founded: 2018 Focus area: In vivo “gene writing” Lead candidate: Multiple preclinical programs Tessera Therapeutics is developing a platform called gene writing, which uses RNA-guided enzymes to make precise edits to DNA directly inside the body. The approach is designed to go beyond traditional gene editing tools like CRISPR by enabling not only cuts but also insertions, deletions, or corrections of DNA sequences, all delivered via lipid nanoparticles (LNPs). At the May 2025 ASGCT meeting, Tessera reported robust preclinical gene editing results in non-human primates: up to 76% correction in liver cells for alpha-1 antitrypsin deficiency and 70% correction for phenylketonuria, with durable effects lasting at least six months and no off-target activity detected. They also showed gene writing in long-term hematopoietic stem cells, achieving more than 20% editing levels, potentially sufficient to treat sickle cell disease. Tessera has raised approximately $610 million to date, including $230 million in series B in 2021, a $300 million series C in 2022, and an additional Gates Foundation investment in 2024. Boston: A powerhouse of biotech innovation The Boston biotech ecosystem is one of the most dynamic and advanced in the world, home to nearly a thousand biotechnology companies and a dense network of research institutions, incubators, and investors. Anchored by academic giants like Harvard and MIT, and completed by world-leading hospitals and research centers, the Boston–Cambridge cluster continues to be a leader in life sciences. This year’s BIO International Convention, held in Boston, only reinforced the city’s pivotal role in shaping the industry’s future. As we discussed in our recent podcast episode, themes like next-gen RNA technologies, artificial intelligence (AI)-assisted discovery, and cell therapy platforms stood out, and many of them are also driven by companies based right here. Additionally, Boston and Cambridge are also home to world-leading U.S. VC firms investing in promising life sciences research. Examples include Third Rock Ventures, RA Capital Management, and of course, Flagship Pioneering, which also acts as a company builder.