What Altheia Science does

Altheia Science is a clinical-stage biotechnology company developing cell and gene therapies for autoimmune diseases, with headquarters in Milan, Italy and a US office in New York City. The company was founded in 2017 as a spin-off from the University of Padua and operates with a lean team of 1–10 employees, indicating an R&D-focused organization built around a single core platform.

The company's technology platform centers on a PD-L1 lentiviral gene therapy delivered into hematopoietic stem and progenitor cells (HSPCs). By restoring PD-L1 expression, the therapy is designed to re-establish immune tolerance in autoimmune settings. Altheia has three named pipeline programs: PD-L1 gene therapy for Type 1 diabetes, PD-L1 gene therapy for multiple sclerosis, and a gene therapy for acute myeloid leukemia (AML). The platform is supported by eight peer-reviewed publications disclosed in 2025, and the company presented its first human-study data in Type 1 diabetes in July 2026, marking the transition into clinical-stage development. Manufacturing is supported through a July 2022 partnership with AGC Biologics.

Altheia's business model is consistent with a venture-backed clinical-stage biotech: it is pre-revenue, with no approved products, and is funded by equity capital rather than product sales. The most recent disclosed financing was a $19.3M (€17M) Series A closed in December 2018, led by Rottapharm Biotech, which remains the anchor institutional investor on record. Future revenue mechanics, once products reach commercialization, will depend on indication-specific pricing, payer dynamics in autoimmune disease, and whether the company pursues direct commercialization or out-licensing to large pharma partners.

Name

Altheia Science

Legal name

Altheia Science S.r.l.

Website

https://altheiascience.com

Company type

Private

Founded year

2017

Operating status

Operating

Headcount range

1–10 employees

Short description

Altheia Science is a Milan-based, clinical-stage biotech developing PD-L1 lentiviral gene therapies for hematopoietic stem cells to treat autoimmune diseases including Type 1 diabetes, multiple sclerosis, and AML.

Ownership category

akta.pro rank

Product category

Gene Therapy / Cell Therapy Biotechnology

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Scientific Research and Development Services (5417)

akta.pro primary industry

Lentiviral Vector Gene Therapy Developers (HLAAACAB)

akta.pro secondary industries

Cell Therapy Developers (Allogeneic/Off-the-shelf) (HLAAABAB), Stem Cell Therapies (MSC/iPSC/HSC-derived) (HLAAABAE), Stem Cell–Based Regenerative Therapies (HLAAAGAA), Neurology/CNS Gene Therapies (HLAAACAH)

Keywords

• Gene therapy
• Cell therapy biotech
• Lentiviral vectors
• Autoimmune disease treatments
• Hematopoietic stem cells

Headquarters

HQ city

Milano

HQ country

Italy

HQ region

Europe

Offices2 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales

Go-to-market motion1 record

Distribution channels1 record

Marketing channels4 records

Core offering

Altheia Science develops lentiviral vector-based gene therapies that restore PD-L1 expression in hematopoietic stem and progenitor cells to induce immune tolerance in patients with autoimmune diseases. Its therapeutic pipeline targets Type 1 diabetes, multiple sclerosis, and acute myeloid leukemia, aiming to provide curative treatments that address the underlying autoimmune mechanism rather than just managing symptoms.

Product overview

Altheia Science is a biotech company developing lentiviral vector-based gene therapies centered on PD-L1 (Programmed death-ligand 1) expression in hematopoietic stem and progenitor cells. Their core technology platform enables restoration of PD-L1 expression, which is lacking or decreased in patients with autoimmune diseases, to overcome autoimmune reactions and promote immune tolerance. The company's therapeutic pipeline includes PD-L1 Gene Therapy for Type 1 Diabetes, PD-L1 Gene Therapy for Multiple Sclerosis (delivered directly into the CNS), and Gene Therapy for Acute Myeloid Leukemia.

Differentiator

Problem solved

Functional benefit

Products and services

• PD-L1 Gene Therapy for Type 1 Diabetes Lentiviral vector-based gene therapy that restores PD-L1 expression in hematopoietic stem and progenitor cells to induce immune tolerance and reverse autoimmune reactions, specifically targeting Type 1 Diabetes. Designed to potentially eliminate the need for exogenous insulin by preserving pancreatic beta-cell function.
• PD-L1 Gene Therapy for Multiple Sclerosis Gene therapy approach delivering PD-L1-expressing hematopoietic stem and progenitor cells directly into the central nervous system to dampen autoimmune mechanisms causing myelin disruption in Multiple Sclerosis.
• Gene Therapy for Acute Myeloid Leukemia Novel gene therapy approach for Acute Myeloid Leukemia targeting newly identified tumour-specific antigens on hematopoietic stem and progenitor cells to overcome limitations of existing targeted therapies.

Quantifiable outcome

• Restoration of PD-L1 expression on hematopoietic stem and progenitor cells successfully reverses Type 1 diabetes in non-obese diabetic mouse models, achieving glucose control normalization and preservation of pancreatic beta-cell function and endogenous insulin production.

Segments1 record

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature1 record

Partnerships

Four partnerships are on record, tiered core and minor.

• AGC BiologicscoreTechnology or Integration · 28 July 2022AGC Biologics supports Altheia Science's gene therapy programs through manufacturing partnership. AGC Biologics is a global CDMO (Contract Development and Manufacturing Organization) providing gene therapy manufacturing capabilities. This partnership enables Altheia Science to advance its PD-L1 gene therapy programs for autoimmune diseases through clinical development.
• University of MilanocoreStrategic or Co-development PartnerAltheia Science is accredited as a spin-off of the University of Milano, indicating a deep research partnership and technology transfer relationship with the university. This affiliation provides access to academic research capabilities and validates the company's biotech credentials.
• University of PadovacoreStrategic or Co-development PartnerAltheia Science is affiliated with the University of Padova as a recognized spin-off, with co-founders and leadership actively participating in university-organized innovation events. The company presented at multiple Padova-based events including Open Innovation Days in collaboration with il Sole 24 Ore.
• Italian Trade AgencyminorGTM or Marketing PartnerPresented at Swiss Biotech Day 2019 within the Italian Trade Agency workshop, indicating support for international market development and promotion of Italian biotech companies abroad.

Scale indicators2 records

Recent moves7 records

Expansion highlights4 records

Direct peers

• Vertex Pharmaceuticals: Vertex is developing VX-880 (zimislecel), a stem cell-derived islet cell therapy for Type 1 Diabetes — the most directly comparable curative T1D program and a benchmark competitor for Altheia's PD-L1 gene therapy approach.
• CRISPR Therapeutics: CRISPR Therapeutics develops ex vivo gene-edited cell therapies using hematopoietic stem cells and has autoimmune disease programs, making it a directly comparable gene therapy platform player.
• Editas Medicine: Editas is a clinical-stage gene editing company developing ex vivo gene-edited cell therapies for serious diseases, including autoimmune indications, with overlapping lentiviral/AAV delivery and HSPC modification platforms.
• Intellia Therapeutics: Intellia is a leading in vivo and ex vivo CRISPR/Cas9 gene editing company with autoimmune disease ambitions; it competes for the same investor capital and partnership channels as Altheia in the cell/gene therapy autoimmune space.
• Sangamo Therapeutics: Sangamo is a pioneer in lentiviral and AAV-based gene therapy, including HSPC-targeted programs, making it a directly comparable lentiviral platform developer with overlapping autoimmune ambitions.
• bluebird bio: bluebird bio commercialized lentiviral ex vivo gene-modified cell therapies (Skysona, Lyfgenia), sharing Altheia's core lentiviral HSPC manufacturing and regulatory pathway challenges.

Emerging players

• Beam Therapeutics: Beam is a base-editing clinical-stage biotech with autoimmune disease programs; partial overlap with Altheia's ex vivo HSPC modification approach but differentiated by base-editing chemistry.
• Tessa Therapeutics: Tessa is developing cell therapy modalities (including CAR-T approaches) for autoimmune diseases; relevant emerging peer in the curative autoimmune cell therapy category alongside Altheia.
• Precision BioSciences: Precision develops in vivo and ex vivo gene-edited cell therapies, including autoimmune programs, providing a relevant comparator in the gene-modified cell therapy platform landscape.

Others

• AGC Biologics: AGC Biologics is Altheia's CDMO partner and a leading lentiviral gene therapy manufacturer; included as an adjacent infrastructure peer that defines manufacturing cost benchmarks and partnership economics for Altheia's platform.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat2 records

Key risks5 records

Key highlights7 records

Customer concentration

Revenue estimate

Valuation estimate

Number of profiles

Profiles6 records

Funding overview

Funding rounds1 record

Investors1 record

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Altheia Science