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.
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.
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.
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.
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.
Gordian Biotechnology
Gordian Biotechnology is a South San Francisco in vivo drug discovery company building a high-throughput screening platform that tests hundreds of gene therapies simultaneously inside animals that naturally develop age-related diseases. By pairing 'patient avatar' models with single-cell transcriptomics and machine learning (a stack the company calls Mosaic Screening + Pythia), Gordian aims to predict human clinical outcomes earlier and cure complex diseases of aging.
Pacira BioSciences, Inc.
Pacira BioSciences (Nasdaq: PCRX) is a U.S. biopharmaceutical company built around a single, stubborn idea: surgery and chronic pain should not require a bottle of opioids. Its flagship product, EXPAREL, a long-acting local anesthetic built on the proprietary DepoFoam multivesicular-liposome delivery platform, anchors a portfolio that also includes the ZILRETTA knee-osteoarthritis injection and the iovera handheld cryoanalgesia device. With roughly 790 employees and about $726 million in 2025 revenue, Pacira is pushing beyond established analgesics into gene therapy (PCRX-201) under a '5x30' growth strategy aimed at reinventing how the world treats pain.
Sangamo Therapeutics, Inc.
Sangamo Therapeutics is a clinical-stage genomic medicine company in Brisbane, California that engineers zinc finger proteins to switch genes on, off, or rewrite them. After three decades pioneering zinc finger technology, it is racing to bring its lead gene therapy ST-920 for Fabry disease to market while building a neurology pipeline aimed at crossing the blood-brain barrier - all under the pressure of a 2026 search for strategic alternatives.
Tune Therapeutics
Tune Therapeutics is a genetic-medicine company building tools to turn genes up or down without cutting DNA. Its TEMPO epigenetic-editing platform tunes gene expression the way a sound engineer rides a fader - reversible, programmable, and precise. Spun out of Duke's Gersbach Lab, Tune raised over $175M in Series B financing in January 2025 and is running first-in-human trials of TUNE-401, an epigenetic silencer for chronic hepatitis B, with the larger ambition of editing the genome's software to extend human healthspan.
Aera Therapeutics
Aera Therapeutics is a Cambridge, Massachusetts biotech founded on a discovery from CRISPR pioneer Feng Zhang: human proteins that can self-assemble into capsid-like shells and ferry genetic cargo into cells. The company is building delivery platforms - protein nanoparticles, targeted lipid nanoparticles, and antibody-oligonucleotide conjugates - to solve the field's most stubborn problem: getting genetic medicines to the right tissue. Launched publicly in 2023 with $193M and led by Alnylam veteran Akin Akinc, Aera is betting that delivery, not the drug itself, is the bottleneck holding back the next generation of genetic medicine.
CellFE
CellFE is an Alameda, California biotechnology company building microfluidics-based, non-viral cell-engineering instruments and consumables. Its Ryva mechanoporation platform squeezes cells through microfluidic channels to transiently open their membranes and deliver gene-editing payloads (mRNA, CRISPR-RNP, DNA vectors) in under 10 milliseconds, aiming to preserve cell health and viability where viral vectors and electroporation fall short. The goal is to make lifesaving cell therapies such as CAR-T faster, cheaper, and more scalable to manufacture.
克睿基因 Cure Genetics
克睿基因 Cure Genetics is a clinical-stage biotech founded in Suzhou in 2016 that develops cell and gene therapies for solid tumors and genetic diseases. It runs two proprietary platforms: AIMS, an allogeneic CAR-NKT cell therapy approach aimed at hard-to-treat solid tumors, and VELP, a directed-evolution AAV vector platform that builds high-quality viral libraries for tissue-targeted gene delivery to the nervous system, heart, and kidney. Its lead programs include CGC729 (anti-CD70 CAR-NKT for renal cell carcinoma) and CGC-602 (a PD-1-IL-2v bispecific antibody).
Kriya Therapeutics, Inc.
Kriya Therapeutics is a clinical-stage biopharmaceutical company building one-time, AAV-based gene therapies for prevalent chronic diseases of high unmet need. Founded in 2019 and headquartered in Morrisville, North Carolina with operations in Silicon Valley, Kriya pairs a proprietary, vertically integrated manufacturing and R&D engine with a pipeline spanning ophthalmology, metabolic disease, and neurology. Its bet: gene therapy shouldn't be reserved for the rare few, but engineered and manufactured at the scale and cost needed to reach millions.