gene-therapy
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The $85 Million Bet That a DNA Scaffold Can Make Gene Therapy Re-Dosable
Code Biotherapeutics is trying to turn DNA into the FedEx network for genetic medicine. Its modular 3DNA scaffold promises bigger payloads, precise addresses and repeat delivery - but the public evidence is still preclinical.
Novartis Cut Away an Empire to Bet on Five Ways of Making Medicine
The Swiss giant spent years shedding businesses until only innovative medicines remained. Now its growth depends on turning five scientific platforms into treatments that health systems will pay for - and patients can actually reach.
Kyowa Kirin, Inc. - U.S.
A Japanese fermentation lineage became a U.S. rare-disease business by following biology that larger markets often overlook. Now Kyowa Kirin is testing whether patient-scale focus can coexist with manufacturing-scale ambition.
Legend Biotech
Legend Biotech turned an experiment from a room the size of a freight elevator into a medicine used to treat more than 10,000 people. Now it must prove that one demanding, personalized therapy can become a platform for the next generation of cancer care.
Nuevocor
Nuevocor is a Singapore-founded, clinical-stage biotechnology company developing genetic medicines for cardiomyopathies driven by aberrant mechanobiology - the mechanical stresses that damage heart-muscle cells. Spun out of Singapore's A*STAR research agency in 2021, the company built a proprietary discovery engine called PrOSIA (Protein Organ Specific Interactome Analysis) to map the functional root causes of genetic heart disease. Its lead program, NVC-001, is an AAV-based gene therapy for LMNA-related dilated cardiomyopathy, an aggressive inherited heart condition with no targeted treatment. Nuevocor received FDA IND clearance for NVC-001 in June 2025 and has raised roughly US$80.6 million to date, including a US$45 million Series B in May 2025.
Chris McNulty
Chris McNulty is the interim CEO of Ring Therapeutics, a Cambridge, Massachusetts biotech developing genetic medicines that use commensal anelloviruses as delivery vectors. An MIT-trained engineer with a Harvard MBA, he joined Ring in May 2023 as Chief Financial Officer and Head of Corporate Strategy and stepped up to lead the company in December 2024. Over more than 15 years in pharma and biotech finance, he has closed financing transactions worth more than $300 million across companies including VBI Vaccines, InVivo Therapeutics, Repligen, and Genzyme.
Beacon Therapeutics
Beacon Therapeutics is a clinical-stage ophthalmic gene therapy company building AAV-based treatments to preserve and restore sight for people with rare and prevalent retinal diseases that cause blindness. Formed in 2023 by Syncona around the assets of Applied Genetic Technologies Corp (AGTC) and complementary science from the University of Oxford, its lead program, laru-zova (AGTC-501), is a one-time gene therapy for X-linked retinitis pigmentosa now in registrational trials, with additional programs targeting geographic atrophy (dry AMD) and cone-rod dystrophy.
Carbon Biosciences
Carbon Biosciences is a Waltham, Massachusetts preclinical genetic-medicine company building gene therapies on non-AAV parvovirus vectors. Founded by Longwood Fund with gene-therapy pioneers John Engelhardt and Robert Kotin, it uses a proprietary vector engine (PAVE) to design capsids from the broad parvovirus family - including human bocavirus - that carry larger genetic payloads, target specific tissues, and provoke little pre-existing immunity, opening the door to re-dosing. Its lead program targets cystic fibrosis by delivering the full-length CFTR gene to the lung, with a second vector aimed at cardiac disease.
Neurotech Pharmaceuticals, Inc.
Neurotech Pharmaceuticals is a Cumberland, Rhode Island biotechnology company that develops long-acting therapies for chronic retinal diseases using its Encapsulated Cell Technology (ECT) platform - a tiny implant of genetically engineered cells that continuously secretes a therapeutic protein inside the eye. In March 2025 its lead product ENCELTO (revakinagene taroretcel-lwey, formerly NT-501/Renexus) became the first and only FDA-approved treatment for macular telangiectasia type 2 (MacTel), delivering ciliary neurotrophic factor (CNTF) to slow the loss of photoreceptors.
Ring Therapeutics
Ring Therapeutics is a Cambridge, Massachusetts biotechnology company founded by Flagship Pioneering to build a new class of gene therapy delivery vectors from anelloviruses - the most abundant and diverse family of viruses that naturally and harmlessly live inside the human body. Because these commensal viruses evade the immune system, Ring's AnelloVector platform aims to solve gene therapy's biggest limitations: the inability to re-dose patients, pre-existing immunity, and narrow tissue targeting. Through its Anellogy discovery platform and AnelloBricks in-vitro manufacturing technology, Ring is engineering re-dosable, tissue-specific, scalable vectors intended to carry a wide range of therapeutic payloads.
Joanna Stanicka
Joanna Stanicka is the co-founder, president and CEO of Axonis Therapeutics, a Boston clinical-stage biotech developing first-in-class oral medicines that target the brain chloride transporter KCC2 to treat epilepsy, neuropathic pain and spinal cord injury. A Harvard-trained neuroscientist who began her career in cancer biology in Ireland, she moved from the bench to the boardroom, taking Axonis from a Boston Children's Hospital discovery to a company that raised a $115 million Series A in 2024 and pushed its lead drug, AXN-027, into first-in-human trials.
Joel Schneider
Joel Schneider is the founder, president and CEO of Carbon Biosciences, a Waltham, Massachusetts biotech building gene therapies from parvovirus-derived vectors rather than the more common adeno-associated viruses. He launched the company out of stealth in 2022 with a $38 million Series A led by Agent Capital, pairing his operating background with the science of gene therapy pioneer Robert Kotin. Trained as a stem cell biologist with a Rutgers Ph.D. and a Harvard postdoc, Schneider was the first employee at Solid Biosciences before founding Carbon to push vectors that carry larger genetic payloads, target specific tissues such as the lung, and sidestep the pre-existing immunity that limits current gene therapies. Carbon's lead program targets cystic fibrosis.
Krystal Biotech, Inc.
Krystal Biotech is a Pittsburgh-based commercial-stage biotechnology company that pioneered redosable, off-the-shelf gene therapy delivered directly to the body. Its lead product VYJUVEK became the first FDA-approved topical and redosable gene therapy in 2023, treating the rare skin disease dystrophic epidermolysis bullosa. Built on a proprietary HSV-1 vector platform (STAR-D), the company runs its own GMP manufacturing and is extending the same delivery technology into respiratory disease, oncology, ophthalmology and aesthetics.
Ansa Biotechnologies, Inc.
Ansa Biotechnologies is an Emeryville, California biotech company that makes custom synthetic DNA using a proprietary enzymatic synthesis platform. Its no-assembly approach can build long, complex, high-fidelity DNA sequences - including clonal constructs up to 50 kilobases - that stump legacy chemical methods, and it backs delivery with an industry-first On-Time Guarantee. Founded in 2018 by UC Berkeley researchers, Ansa serves scientists across pharmaceuticals, gene therapy, agriculture, and industrial biotech.
Cyagen Biosciences
Cyagen Biosciences is a life-sciences contract research organization that builds custom genetically engineered rodent models - knockout, knockin, conditional, and humanized mice and rats - for academic labs and drug developers. Founded in 2006 and headquartered in Santa Clara, California, with major operations in China, Japan, and Europe, Cyagen has grown into one of the world's largest suppliers of custom mouse and rat models, complemented by stem-cell products and a fast-growing gene-delivery arm, VectorBuilder, that designs and manufactures viral vectors from research through clinical CDMO work.
Dispatch Bio
Dispatch Bio is a Philadelphia-based cancer biotech that emerged from stealth in July 2025 with $216 million to pursue a 'universal' immunotherapy for solid tumors. Its first-in-class Flare platform uses an engineered virus to install a synthetic, tumor-specific antigen on cancer cells while remodeling the tumor's immune-suppressive environment, effectively giving CAR T cells a target where none existed. Founded in 2022 out of the Parker Institute for Cancer Immunotherapy and ARCH Venture Partners, the company is led by CEO Sabah Oney and counts CAR T pioneer Carl June among its scientific co-founders.
KaliVir Immunotherapeutics
KaliVir Immunotherapeutics is a clinical-stage biotech developing next-generation oncolytic viral immunotherapies for cancer. Its proprietary Vaccinia Enhanced Template (VET) platform engineers vaccinia viruses that can be delivered intravenously, replicate selectively in tumors, and express therapeutic payloads that reshape the tumor microenvironment. Founded in 2019 and headquartered in Pittsburgh, the company advances an internal lead candidate, VET3-TGI, alongside partnered programs licensed to Astellas Pharma and Roche.
SonoThera
SonoThera is a South San Francisco biotech developing an ultrasound-guided, nonviral platform for delivering genetic medicines. Instead of the viral vectors that dominate gene therapy, it uses focused ultrasound and injectable microbubbles - a process called sonoporation - to open transient pores in target cells and shuttle DNA or RNA payloads into specific organs. The pitch is delivery that is targeted, potentially redosable, and decoupled from the immune and manufacturing headaches of viruses. The company raised a $125M Series B in June 2026 and is pushing lead programs in Duchenne muscular dystrophy and polycystic kidney disease toward the clinic.
T-knife Therapeutics
T-knife Therapeutics is a clinical-stage biotech engineering T-cell receptor (TCR-T) therapies to treat solid tumors. Spun out of Berlin's Max Delbrück Center in 2015, it built a proprietary humanized-TCR mouse platform (HuTCR) that grows a full human immune repertoire in mice, letting the company harvest fully human TCRs naturally selected for affinity and specificity. Now dual-based in San Francisco and Berlin, T-knife is advancing 'supercharged' TCR-T candidates - including a PRAME-targeted, CRISPR-armored therapy (TK-6302) entering the clinic - against cancers that have long resisted cell therapy.
Rich Small
Rich Small is the CEO of Neurotech Pharmaceuticals, the Cumberland, Rhode Island clinical-stage biotech that in March 2025 won FDA approval for ENCELTO, the first and only treatment for macular telangiectasia type 2 (MacTel). A CPA and Northeastern University graduate, he joined Neurotech as CFO in 2007 and took over the top job in 2016, guiding the company's encapsulated cell technology platform from experimental science to a commercial retinal implant.
Syed T. Husain
Syed T. Husain is the Chairman and CEO of Made Scientific, a Princeton, New Jersey cell therapy contract development and manufacturing organization (CDMO) that he relaunched in 2025 out of the former BioCentriq. Backed by GC Corporation, the company runs a 60,000-square-foot GMP facility built to move autologous and allogeneic cell therapies from clinical batches to commercial supply. A chemical engineer by training who started on the manufacturing floor at Wyeth and Pfizer, Husain spent more than two decades in CDMO leadership at Lonza, Alcami, Emergent BioSolutions, and Resilience before taking the top job. He named the rebranded company MADE for Manufacturing and Development Excellence.
Tuyen Ong
Tuyen Ong is a physician-turned-biotech executive who spent four years as CEO of Ring Therapeutics, the Flagship Pioneering company trying to turn anelloviruses - harmless viruses that already live in nearly everyone - into a redosable delivery system for genetic medicines. Trained as an ophthalmologist at University College London with an MBA from NYU Stern, he built his career on gene therapy for rare eye disease at Nightstar, Biogen, PTC, Bausch & Lomb and Pfizer before betting on a virus most scientists had written off as biological junk. In 2025 he co-founded PharmasMarket, a drug-development consultancy.
Xuandai Nguyen
Xuandai Nguyen, Ph.D., MBA, is Vice President, Head of Global Program Strategy and Chief of Staff to the CEO at Beacon Therapeutics, an ophthalmic gene therapy company working to restore sight in people with inherited and prevalent retinal diseases. A scientist-turned-strategist, Nguyen has spent more than two decades moving medicines from bench to market, with senior roles at Genentech, Forma Therapeutics and ACELYRIN spanning immunology, ophthalmology, respiratory and sickle cell disease. Trilingual and Berkeley- and Cornell-trained, Nguyen sits at the intersection of hard science and commercial strategy, translating complicated biology into the plans that decide which therapies reach patients.
Axelyf
Axelyf is a Brookline, Massachusetts biotech (with an R&D subsidiary in Iceland) building next-generation lipid nanoparticle delivery systems for RNA medicines. Founded by former Moderna drug-delivery leaders, it pairs a proprietary AXL ionizable-lipid library with an AI model called ANNA to make RNA and gene-editing payloads reach the right tissue with higher potency and less toxicity. In mouse liver studies its AXL LNPs showed roughly 4-5x higher gene-editing efficiency than the LP-01 clinical benchmark at equivalent doses, without evidence of liver toxicity. The company closed $2.6M in seed funding in August 2025 to push its lead autoimmune program and lipid library toward preclinical and non-human-primate studies.
GEMMA Biotherapeutics
GEMMA Biotherapeutics (GEMMABio) is a Philadelphia-based gene therapy company spun out of the University of Pennsylvania and founded by AAV pioneer James M. Wilson. Launched in October 2024 with $34 million in seed funding, GEMMABio builds the research, manufacturing, and product-development infrastructure to move adeno-associated virus (AAV) gene therapies from the bench to the bedside faster and more affordably - with a focus on rare and ultra-orphan inherited diseases and a stated commitment to global patient access.
Grace Science, LLC
Grace Science, LLC is a Palo Alto-based biotechnology company founded in 2017 by Silicon Valley entrepreneur Matt Wilsey and Nobel laureate chemist Dr. Carolyn Bertozzi to develop therapies built on the biology of NGLY1, an enzyme central to protein quality control. The company grew out of a father's search for a treatment after his daughter Grace was diagnosed with ultra-rare NGLY1 Deficiency. Its lead program, GS-100, is an AAV9 gene replacement therapy now in a Phase 1/2/3 clinical trial and holds FDA RMAT designation. Grace Science also pursues small-molecule and biomarker work that extends NGLY1 biology into oncology and neurodegeneration.
K2bio
K2bio is a Houston-based preclinical contract research organization (CRO) built on assets acquired from Bellicum Pharmaceuticals, including a certified mouse vivarium. It pairs full-service GLP and non-GLP in vivo and in vitro research with flexible private and shared lab space near the Texas Medical Center, giving early-stage biotech and pharma teams big-company infrastructure on small-company budgets.
Tiba Biotech
Tiba Biotech is a Cambridge, Massachusetts preclinical biotech spun out of MIT and the Whitehead Institute that is building a new generation of RNA vaccines and therapeutics. Its RNABL platform swaps the fragile lipid nanoparticles behind first-generation mRNA shots for fully synthetic, biodegradable dendrimer nanoparticles that carry larger RNA payloads with less toxicity and gentler cold-chain demands. The company is applying the technology across human vaccines, cancer immunotherapy, gene editing delivery, and animal health, backed by CEPI, BARDA, the NIH, and the NCI.
Jack Milwid
Jack Milwid is a scientist-turned-company-builder who sits in the Office of the CEO at Lila Sciences, the Flagship Pioneering venture chasing 'scientific superintelligence' through autonomous AI-run laboratories. A materials engineer with a Harvard-MIT PhD, he has spent his career founding biotech companies from scratch - among them Sana Biotechnology, Neon Therapeutics, Cobalt Biomedicine and Kaleido Biosciences - and co-founded the investment firm Related Sciences. His trademark is turning early science into operating companies.
Amber Bio
Amber Bio is a biotechnology startup pioneering a new RNA writing platform that performs multi-kilobase edits, aiming to treat thousands of disease-causing mutations with a single product. Founded in 2021 by Jacob Borrajo and Basem Al-Shayeb out of the Broad Institute and UC Berkeley, the company launched in August 2023 with a $26M seed round co-led by Playground Global and Andreessen Horowitz Bio + Health.