AlphaRose Therapeutics

AlphaRose Therapeutics is a groundbreaking precision medicine company focused on advancing genetic treatments with our ASO platform and AI. Built upon the pioneering work of industry leaders like Henri Termeer and inspired by the incredible stories of Milasen and others.

Our mission is to revolutionize the delivery of genetic therapies, speeding up their availability, lowering risk, and expanding access, starting with children who need them the most. Our team of scientists, biotech professionals, and parents are united by a shared purpose: to create life-changing medicines for children suffering from genetic diseases.

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Apertura Gene Therapy

Gene therapy company that has engineered a capsid targeting the CNS dosed IV. We have three programs using our capsid that will be in the clinic in the next 12 months.

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CDG CARE

CDG CARE (Community Alliance and Resource Exchange) is a 501(c)(3) nonprofit public charity dedicated to representing all Congenital Disorders of Glycosylation (CDG) — a group of rare, inherited metabolic disorders that disrupt a complex enzymatic process essential to nearly every system in the body.

Founded by parents seeking information, connection, and hope, CDG CARE has grown into a global organization serving patients, families, clinicians, and researchers across more than 200 known CDG types.

Our mission is to raise awareness, support families, and fund research to improve the diagnosis, care, and treatment of CDGs. We pursue this mission through programs that meet families wherever they are in their journey, from the moment of diagnosis through the search for therapies.

At every level of our work, CDG CARE is driven by a simple belief: that no family facing a CDG diagnosis should feel alone, and that together… families, scientists, clinicians, and advocates, we can move research, therapies, and hope forward.

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Citizen Health

Citizen Health is building the future of healthcare, starting with rare and complex diseases. Its AI teammate, Ari, helps patients and caregivers interpret medical records, track symptoms, manage appointments and insurance, and connect to the next best step in their care. Citizen Health is headquartered in San Francisco, California.

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Coriell Institute for Medical Research

The Coriell Institute for Medical Research, originally known as the South Jersey Medical Research Foundation and then as the Institute for Medical Research, was founded by Dr. Lewis L. Coriell in 1953. Dr. Coriell, a Physician and a Virologist was a scientific pioneer, making invaluable contributions to biomedical research through advances in aseptic cell culture techniques, cryopreservation of cell lines, and clinical trials of polio prevention.

Over the past 72 years, the Coriell Institute has grown into a world-renowned biomedical research institute. In pursuit of our mission to prevent and cure disease, Coriell scientists conduct research in rare diseases, cancer, aging, infectious diseases, and personalized medicine. Coriell is one of the top recipients of research awards from the National Institutes of Health (NIH) in New Jersey. Coriell was one of the first two official cell banks recognized by the NIH in 1960 and has served as an NIH-supported cell repository for more than 50 years, currently hosting biobanks for the following NIH institutes: NIGMS, NINDS, NHGRI, NEI, and NIA. Coriell also provides its laboratory and biobanking expertise to researchers and organizations who require assistance processing, storing and distributing their samples.

In partnership with Cooper University Health Care and the Cooper Medical School at Rowan University, Coriell established the Camden Cancer Research Center (CCRC) in 2023, the first basic cancer research center in southern New Jersey. In 2025, in partnership with the New Jersey Economic Development Authority (NJEDA), Coriell announced the formation of a new state-designated Strategic Innovation Center (SIC) to support emerging biotech companies. The New Jersey Coriell Labs Innovation Center SIC will be the largest public life sciences incubator in the region and serve as the focal point to spur collaboration among the region’s academic, healthcare and research institutions.

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Dyno Therapeutics

We are AI and biotech pioneers powering genetic agency, an individual’s ability to take action at the genetic level to live a healthier life. We build high-performance genetic technologies to unlock the potential of next-gen medicine and enable genetic agency for all patients.

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The Jackson Laboratory

The Rare Disease Translational Center (RDTC) at The Jackson Laboratory (JAX) partners with patient families, foundations, academic investigators, and biotechnology companies to accelerate the development of therapies for rare diseases. The RDTC specializes in the development and characterization of high-quality mouse and cellular disease models of rare and ultra-rare disorders. These models serve as critical tools for understanding disease biology, defining mechanisms of pathology, identifying therapeutic targets, and evaluating potential treatments. We support projects across the entire translational research pipeline, from early-stage disease modeling and target validation to biomarker discovery, preclinical efficacy studies, and therapeutic development strategies. Our multidisciplinary team works closely with collaborators to design studies that answer the most important scientific and translational questions, providing tailored solutions that align with the needs of each program. Building on more than a century of knowledge in genetics and biomedical research, the RDTC brings together deep expertise in genetics, genomics, model engineering, phenotyping, preclinical study design, and translational science. We have extensive experience supporting a broad range of therapeutic modalities, including gene replacement therapies, genome editing approaches, antisense oligonucleotides (ASOs), RNA-based therapeutics, and small molecules. Together, we help turn genetic discoveries into therapeutic opportunities and therapeutic opportunities into hope for patients and families.

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Nome

Nome is for rare disease families, foundations, and health systems. We are a services company that helps them access and develop personalized genetic medicines. We work across the full lifecycle of a therapeutic program — from treatment design to clinical execution — using an AI platform combined with human experts to make this process faster, lower-cost, and more accessible than traditional drug development. For families and organizations navigating the emerging world of n-of-1 and rare disease genetic therapies, we are the ‘easy button’ that provides concierge navigation through this process. Nome is the operating system for personalized therapies.

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Unravel Biosciences

The rareSHIFT™ program is Unravel Biosciences’ personalized medicine program, providing rare, ultra-rare, and undiagnosed disease patients with individualized treatment insights generated using AI and each individual’s primary patient data. The program recognizes the massive need for therapeutics in the rare disease space. No patient’s disease presentation is exactly the same as another’s. Treatments intended for the average presentation of a disease will inevitably leave many patients underserved. rareSHIFT™ addresses this gap by generating therapeutic predictions for each individual patient based on mechanistic insights from their unique RNA data.

The program is powered by Unravel’s Predictable Medicine™ development platform, which starts with each patient’s own primary data. Using a non-invasive, at-home nasal swab collection method, patients anywhere in the world can contribute RNA transcriptomic data without requiring specialized clinical infrastructure. From these samples, RNAseq data is generated to construct a Living Molecular Twin™: a digital model of that patient’s individual disease biology. This patient-specific model is central to how rareSHIFT™ differs from conventional drug development approaches. Unravel’s proprietary BioNAV™ platform screens each patient’s Living Molecular Twin™ against a library 40,000+ existing molecules, including FDA-approved drugs, GRAS compounds, and nutraceuticals. The output is a report containing a ranked list of molecules predicted to be therapeutic and antitherapeutic to that patient’s specific biology, and associated information on mechanisms of action, relevant pathways, and more, to help narrow down the best candidates to pursue.

For patient communities, this approach offers a rapid path to actionable therapeutic hypotheses. rareSHIFT™ can identify drug repurposing candidates significantly faster than traditional R&D timelines. Findings from the platform can inform observational studies, clinical trial design, and, in select cases, treatment discussions and decisions led by the patient’s treating clinicians. This program has supported more than 25 clinical successes to date.

rareSHIFT™ is designed to operate in partnership with patient advocacy groups, families, clinicians, and healthcare institutions to syndicate drug development and defragment rare disease. Unravel currently co-develops programs with patient advocacy groups across a range of rare and ultra-rare conditions, pairing each organization’s unique understanding of its patient community with a platform capable of translating individual patient data into therapeutic opportunities.

For communities that are often too small or too dispersed to be prioritized by conventional pharmaceutical development models, rareSHIFT™ offers a scalable mechanism for pursuing treatment options tailored to the patients living with the disease, not an average approximation of it. This approach has resulted in five development programs at Unravel, including two clinical trials, for Rett syndrome and Pitt Hopkins syndrome.

Organizations interested in exploring a rareSHIFT™ partnership can find more information at www.rareSHIFT.org or by emailing [email protected].

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Viragen

Viralgen is a leading CDMO specializing exclusively in AAV-based gene therapies, offering end-to-end support from early development to commercial production. Using our proprietary Aava™ suspension platform, we achieve scalable manufacturing across all AAV serotypes. With cGMP-certified facilities in San Sebastián, Spain, featuring bioreactors up to 2,000L, we provide integrated services including plasmid production, process optimization, fill-finish, and in-house QC testing. Our approach accelerates development timelines while controlling cost-of-goods. With over 1,500 AAV batches produced and a strong regulatory track record, Viralgen delivers reliable, efficient solutions that help bring gene therapies to patients with greater speed and consistency.

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