SCIDNET · Project

Gene Therapy Becoming Licensed Medicine for Children Born Without Immune Systems

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Some babies are born with immune systems that simply don't work — without treatment, they can't survive their first year. Think of it like being born without any armor in a world full of threats. Over 15 years, scientists have developed a way to fix the broken gene responsible, essentially giving these children a working immune system using their own corrected stem cells. SCIDNET pushed this treatment from lab success toward becoming an approved medicine, covering over 80% of all forms of this condition across Europe.

By the numbers

80%

of all SCID forms in Europe covered by the gene therapy programme

15 years

of prior gene therapy development for SCID

forms of SCID addressed (SCID-X1, ADA SCID, plus 3 others with proof of concept)

EUR 6,926,313

EU contribution to the project

consortium partners across 6 countries

total project deliverables

The business problem

What needed solving

Children born with severe combined immunodeficiency have no functioning immune system and face death within their first year without treatment. Current options are limited to bone marrow transplants from matched donors, which are not always available and carry significant risks. There is a critical unmet need for a reliable, curative treatment that can be manufactured and delivered as a licensed medicine across Europe.

The solution

What was built

SCIDNET developed clinical-grade gene therapy protocols for 5 forms of SCID, initiated clinical trials for the most advanced product (ADA SCID with Orphan Drug Designation), and produced 13 deliverables including clinical trial infrastructure and next-generation safety improvements. The project established a regulatory pathway through direct engagement with the European Medicines Agency toward marketing authorisation.

Audience

Who needs this

Gene therapy and biotech companies seeking late-stage rare disease assets to licenseOrphan drug developers looking for products with existing regulatory designationsCDMOs specializing in cell and gene therapy manufacturingRare disease patient advocacy organizations seeking treatment accessHospital networks and paediatric immunology centres treating SCID patients

Business applications

Who can put this to work

Pharmaceutical & Gene Therapy Manufacturing

enterprise

Target: Biotech companies developing advanced therapy medicinal products (ATMPs)

If you are a biotech or pharma company looking to enter the gene therapy market — SCIDNET developed clinical-grade gene therapy protocols for 5 forms of SCID, with one product (ADA SCID) carrying Orphan Drug Designation and a pathway to marketing authorisation through the European Medicines Agency. The consortium of 12 partners across 6 countries built the clinical and regulatory groundwork that a manufacturing partner could leverage to bring these therapies to market.

Rare Disease Therapeutics

mid-size

Target: Orphan drug developers and rare disease-focused companies

If you are a rare disease company struggling with the high cost and complexity of developing treatments for small patient populations — SCIDNET addressed over 80% of all SCID forms in Europe through a single coordinated programme. The ADA SCID product already holds Orphan Drug Designation, meaning regulatory incentives like market exclusivity are in place. This de-risks the commercial pathway significantly for a licensing partner.

Cell & Gene Therapy Services

mid-size

Target: Contract development and manufacturing organizations (CDMOs) for cell therapies

If you are a CDMO looking to expand your gene therapy manufacturing capabilities — SCIDNET developed processes for autologous stem cell gene therapy across multiple disease variants, generating 13 deliverables including clinical trial protocols. The project investigated future technologies to improve safety and efficacy, creating manufacturing know-how that could be licensed or contracted for broader bone marrow disorder applications.

Frequently asked

Quick answers

What would it cost to license or commercialize this gene therapy?

The project received EUR 6,926,313 in EU funding across 12 partners over 4 years, covering research through clinical trial initiation. Commercialization costs would include Phase III trials and manufacturing scale-up, but the Orphan Drug Designation provides regulatory fee reductions and market exclusivity incentives. Specific licensing terms would need to be negotiated with University College London as coordinator.

Can this be manufactured at industrial scale?

The project focused on translating gene therapy from early clinical trials toward a licensed medicinal product, with specific attention to developing the technologies and processes needed for authorisation. Based on available project data, the manufacturing processes were designed for clinical-grade production, but full commercial-scale manufacturing would require a CDMO partnership.

What is the IP and licensing situation?

The ADA SCID gene therapy product holds Orphan Drug Designation under EU regulation, which typically grants 10 years of market exclusivity. University College London coordinates the IP from this 12-partner consortium across 6 countries. Licensing discussions would need to account for contributions from 3 industrial partners and 1 SME in the consortium.

What regulatory approvals are in place?

The lead ADA SCID programme has Orphan Drug Designation and clinical trial design was developed with engagement from the European Medicines Agency. The project initiated clinical trials as confirmed by deliverables, working toward marketing authorisation as a licensed medicine.

How large is the market for SCID gene therapy?

SCID is classified as a rare disease under EU criteria (EC regulation No. 141/2000). While patient numbers are small, orphan drugs command premium pricing — often EUR 200,000-500,000 per treatment — and the project covers over 80% of all SCID forms in Europe. Based on available project data, the technologies developed could also extend to other bone marrow disorders, expanding the addressable market.

What is the timeline to market?

The project ran from 2016 to 2019 and achieved clinical trial initiation. Based on available project data, the ADA SCID product was on a pathway toward marketing authorisation. Post-project, further clinical trials and regulatory review would be needed, but the 15 years of prior gene therapy development provide a strong foundation.

Consortium

Who built it

SCIDNET brings together 12 partners from 6 countries (UK, Germany, France, Italy, Netherlands, Switzerland), led by University College London — one of Europe's leading gene therapy research centres. The consortium includes 4 universities, 2 research organisations, and 3 industrial partners (including 1 SME), giving it a 25% industry ratio. This mix signals serious translational intent: the academic partners provide deep scientific expertise built over 15 years of SCID gene therapy work, while the industrial partners bring manufacturing and commercialisation capability. The EUR 6,926,313 budget and engagement with the European Medicines Agency confirm this is not basic research — it is a clinical-stage programme actively pursuing marketing authorisation.

UNIVERSITY COLLEGE LONDONCoordinator · UK
ACADEMISCH ZIEKENHUIS LEIDENparticipant · NL
PROTAGENE CGT GMBHparticipant · DE
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALEparticipant · FR
MILTENYI BIOTEC GMBHparticipant · DE
MEDIZINISCHE HOCHSCHULE HANNOVERparticipant · DE
CENTRE HOSPITALIER UNIVERSITAIRE VAUDOISparticipant · CH
GREAT ORMOND STREET HOSPITAL FOR CHILDREN NHS FOUNDATION TRUSTparticipant · UK
OSPEDALE SAN RAFFAELE SRLparticipant · IT
UNIVERSITAETSKLINIKUM FREIBURGparticipant · DE
ASSOCIATION GENETHONparticipant · FR

How to reach the team

University College London, UK — gene therapy research group. SciTransfer can help identify the right contact.

Order a report on this consortium See UNIVERSITY COLLEGE LONDON profile →

Next steps

Talk to the team behind this work.

Want to explore licensing opportunities for SCID gene therapy or related bone marrow disorder treatments? SciTransfer can connect you with the SCIDNET research team and provide a detailed technology brief.

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