3rd Spring School OXFORD UK

123 days

4 hours

28 mins

Our Partners

We are very grateful for support from our partners, please click on any logos below to head to their website.

If you would like to see your logo here, head over to our Sponsorship Guide page to find out how we can work together!

Diamond

Merck

As part of Merck KGaA, Darmstadt, Germany, BioReliance® Services is a key component of the life science services portfolio and the leading provider of contract services in the biopharmaceutical industry. BioReliance’s comprehensive and integrated services support every phase of the testing, development and manufacturing process. With locations worldwide, BioReliance offers more than 1,000 tests and complementary services related to biologics safety testing and specialized toxicology. Our clients include the world’s top producers of traditional pharmaceuticals, medical devices and chemicals, as well as biopharmaceuticals

AveXis

“Based in Chicago, Illinois, AveXis is a clinical-stage gene therapy company dedicated to developing and commercializing novel treatments for patients suffering from rare and life-threatening neurological genetic diseases. 
Our initial product candidate, AVXS-101, is our proprietary gene therapy product candidate currently in a Phase 1 clinical trial for the treatment of spinal muscular atrophy (SMA) Type 1, the leading genetic cause of infant mortality. SMA Type 1 is a lethal genetic disorder characterized by motor neuron loss and associated muscle deterioration, resulting in mortality or the need for permanent ventilation support before the age of two for greater than 90% of patients-
We are passionately committed to moving gene therapies into the clinical setting for patients and families devastated by rare and orphan neurological genetic diseases. With the support of industry and academic alliances, we're advancing cutting-edge science in order to treat rare and life-threatening genetic diseases—starting with our clinical-stage, proprietary gene therapy candidate, AVXS-101.”

Platinum

Bluebird Bio

Bluebird bio is developing innovative gene therapies for severe genetic disorders. At the heart of bluebird bio’s product creation efforts is its broadly applicable gene therapy platform for the development of novel treatments for diseases with few or no clinical options. The company’s novel approach uses stem cells harvested from the patient’s bone marrow into which a healthy version of the disease causing gene is inserted.

Oxford Biomedica

Oxford BioMedica (LSE:OXB) is a leading gene and cell therapy company focused on developing life changing treatments for serious diseases. The Company has built a sector leading lentiviral vector delivery platform (LentiVector®) through which it continues to develops in vivo and ex vivo gene & gene-modified therapies products both in-house and with partners. Oxford BioMedica has entered into a number of key partnerships, including with Novartis, Sanofi, GSK, Green Cross Lab Cell and Immune Design. Oxford BioMedica has world class facilities and capabilities encompassing the full range of GMP manufacturing and analytical activities to support pre-clinical, research and bioprocessing development through to GMP production and supply of clinical trial materials.  The production activities are focussed on the manufacture of lentiviral vectors from human cell lines, both in adherent and large scale serum free suspension culture. Oxford BioMedica is based across several locations in Oxfordshire, UK and employs more than 230 people.

Biogen

Through cutting-edge science and medicine, Biogen discovers, develops and delivers to patients worldwide innovative therapies for the treatment of neurodegenerative diseases, hematologic conditions and autoimmune disorders. Founded in 1978, Biogen is one of the world’s oldest independent biotechnology companies and patients worldwide benefit from its leading multiple sclerosis and innovative hemophilia therapies

Voyager Therapeutics

Voyager Therapeutics is a clinical-stage gene therapy company developing life-changing treatments for severe diseases of the central nervous system. Voyager is committed to advancing the field of AAV gene therapy through innovation and investment in vector engineering and optimization, manufacturing and dosing and delivery techniques. The company’s pipeline is focused on severe CNS diseases in need of effective new therapies, including advanced Parkinson’s disease, a monogenic form of amyotrophic lateral sclerosis (ALS), Friedreich’s ataxia, and Huntington’s disease.

Molmed

MolMed S.p.A. is a medical biotechnology company focused on research, development and clinical validation of novel anticancer therapies. MolMed’s pipeline includes antitumour therapeutics in clinical and preclinical development: Zalmoxis® (TK), a cell-based therapy; NGR-hTNF, a novel therapeutic agent for solid tumours; CAR-CD44v6, an immuno-gene therapy project; MolMed also conducts cell and gene therapy projects in collaboration with third parties, offering resources and expertise covering preclinical to Phase III trials activities. MolMed is listed on the main market (MTA) of the Milan stock exchange managed by Borsa Italiana

Adverum

Adverum is a gene therapy company committed to discovering and developing novel medicines that can offer life-changing benefits to patients living with rare diseases or diseases of the eye who currently have limited or burdensome treatment options. Adverum has a robust pipeline and is leveraging its next-generation adeno-associated virus (AAV)-based directed evolution platform to generate product candidates designed to provide durable efficacy by inducing sustained expression of a therapeutic protein. Our focus on the patient is supported by clinical development expertise and core capabilities in vector optimization, process development, manufacturing, and assay development. 

Spark

Spark Therapeutics, a fully integrated company, strives to challenge the inevitability of genetic disease by discovering, developing, and delivering gene therapies that address inherited retinal diseases (IRDs), neurodegenerative diseases, as well as diseases that can be addressed by targeting the liver, such as hemophilia. Spark Therapeutics has ongoing clinical trials investigating gene therapies in hemophilia A and B. SPK-8011 is in an ongoing, dose-escalation Phase 1/2 clinical trial as a potential one-time therapy for hemophilia A. The company retains full global commercialization rights to the SPK-FVIII program. SPK-9001, which has received both breakthrough therapy and orphan product designations by FDA, and access to the PRIority MEdicines (PRIME) Program by EMA, is in a Phase 1/2 clinical trial for hemophilia B and is being developed in collaboration with Pfizer. Our most advanced investigational candidate, with proposed trade name LUXTURNA™ (voretigene neparvovec), is currently under Priority Review with FDA for the treatment of biallelic RPE65-mediated IRD and has been designated as a drug for a rare pediatric disease. The MAA for LUXTURNA has been submitted to EMA for the treatment of vision loss due to Leber congenital amaurosis or retinitis pigmentosa caused by confirmed biallelic RPE65 mutations. LUXTURNA has received breakthrough therapy and orphan product designations from FDA and orphan product designations from EMA. The pipeline also includes SPK-7001 in an ongoing Phase 1/2 clinical trial for choroideremia. 

Gold

Genethon

Genethon, created by AFM-Telethon, has the mission to make innovative  gene therapy treatments available to patients affected with rare genetic diseases. To meet this challenge Genethon has assembled the technical and human resources needed to accelerate the medical application of scientific discoveries arising from fundamental research. Strong translational research programs engage multi-disciplinary teams and are supported by a first rate technological platform and cGMP facility

The pipeline of Genethon includes products currently in international clinical trials and at preclinical stages, for muscular dystrophies, immune deficiencies, blood, ocular and liver diseases. These products are developed either with Genethon as sponsor, or in partnership with private companies and academic institutions.

uniQure

uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with CNS, liver/metabolic and cardiovascular diseases

Sangamo

Sangamo BioSciences (Nasdaq: SGMO) Sangamo BioSciences, Inc. is focused on the development of genetic therapies based on its zinc finger protein (ZFP) technology platform for genome editing and gene regulation, and its AAV-cDNA gene therapy platform. In 2016, the Company expects to initiate a Phase 1/2 clinical trial for its zinc finger nuclease (ZFN)-based therapeutic for the treatment of hemophilia B, which represents the first in vivo genome editing application in man. Sangamo also plans to file an Investigational New Drug (IND) application to initiate a Phase 1/2 clinical trial for hemophilia A based on its AAV-cDNA gene therapy approach. In addition, the Company is developing ZFN-based therapeutics for lysosomal storage disorders, including MPS I (Hurler syndrome) and MPS II (Hunter syndrome), and has strategic collaborations with Biogen Inc. to develop therapeutics for sickle cell disease and beta-thalassemia, and with Shire International GmbH for Huntington’s disease. For more information about Sangamo, visit the Company’s website

Editas

Editas Medicine is a transformative genome editing company founded by world leaders in the fields of genome editing, protein engineering, and molecular and structural biology, with specific expertise in CRISPR/Cas9 and TALENs technologies. The company's mission is to translate its genome editing technology into a novel class of human therapeutics that enable precise and corrective molecular modification to treat the underlying cause of a broad range of diseases at the genetic level. The company has generated substantial patent filings and has access to intellectual property covering foundational genome editing technologies, as well as essential advancements and enablements that will uniquely allow the company to translate early findings into viable human therapeutic products.

MeiraGTx

MeiraGTx is committed to the development of novel gene therapies to transform the lives of patients suffering from acquired and inherited disorders.

We are pioneering the use of gene therapy to treat devastating neurodegenerative diseases such as Alzheimer’s disease (AD), Parkinson’s disease (PD), and amyotrophic lateral sclerosis (ALS). We are developing innovative gene therapies for ocular diseases including rare inherited blindness and age-related macular degeneration (AMD). MeiraGTx is also establishing treatments for xerostomia, a frequent and debilitating side effect of radiation treatment used in head and neck cancers. Our revolutionary gene regulation technologies promise to transform the way gene therapy can be applied and create new paradigms for biologic therapeutics.

 

Maxcyte

MaxCyte's non-viral delivery platform allows for engineering of nearly all cell types, including human primary cells, with any molecule, at any scale for use in drug discovery and development, biomanufacturing, gene editing, cell therapy, and immuno-oncology. Its consistency and minimal cell disturbance facilitate rapid, clinical and commercial grade cell engineering.

Max-Delbrück-Centrum für Molekulare Medizin

The Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC) carries out basic biomedical research with the aim of understanding the molecular basis of health and disease, and translating these findings as quickly as possible into clinical application. These core activities are pursued in more than 60 independent research groups across four main research areas: Cardiovascular and Metabolic Disease, Cancer Research, Diseases of the Nervous System and Medical Systems Biology. As a testament to its success, the MDC has been ranked 14th in the Thompson Reuters list of the world’s 20 best research institutes for molecular biology and genetics, based on our publication record.

Rocket

Rocket Pharma is a clinical-stage gene therapy company with a platform approach for the treatment of patients with high-unmet need rare diseases. We leverage lentiviral and AAV transduction strategies towards first-in-class programs.  Hallmarks of Rocket’s vision include: 1) a high threshold for the selection of quality programs, 2) leverage of deep industry know-how through a world-class team (Novartis Cell & Gene, Bluebird, BMS, others) and manufacturing partnerships (MolMed, PCT, others) and 3) a laser focus on optimizing gene therapy product parameters through a seasoned scientific approach that de-risks programs as they enter clinical trials.

ziopharm

ZIOPHARM Oncology, Inc. (ZIOP) is a Boston, Massachusetts-based biotechnology company employing novel gene expression, control and cell technologies to deliver safe, effective and scalable cell-based therapies for the treatment of cancer and graft-versus-host-disease (GvHD). The Company's synthetic immuno-oncology programs, in collaboration with Intrexon Corporation (NYSE:XON), Merck KGaA (ZIOP, CAR-only), and MD Anderson Cancer Center, include engineered virus for direct in vivo application, chimeric antigen receptor-modified T cells, T-cell receptor-modified T cells, regulatory T cells, and natural killer cells. We use our non-viral- and viral-based gene transfer methods to achieve broad scalability while focusing on managing cost and instilling control of gene therapies. Thus, our cell-based approach is based on rapidly generating products from patients (autologous) and third-party (allogeneic) donors for off-the-shelf therapy. This translates into a preclinical and clinical pipeline to evaluate the treatment in hematological and solid tumor malignancies, as well as GvHD. The Company’s genetic engineering tools include Intrexon's proprietary gene control approaches such as its RheoSwitch® platform and kill switch to advance programs in multiple stages of development, including a Phase III asset.

Silver

TiGenix

TiGenix is a leading European cell therapy company with a commercial product and a strong clinical stage pipeline of adult stem cell programs. The company?s lead product, ChondroCelect®, for cartilage repair in the knee, is the only approved cell-based product in Europe, and is currently being launched across Europe. TiGenix?s adipose derived allogeneic stem cell platform has been extensively validated. TiGenix is based out of Leuven (Belgium) and has operations in Madrid (Spain), and Sittard-Geleen (the Netherlands).

AFM

AFM (French Muscular Dystrophy Association) has a single objective: to defeat neuromuscular diseases, which are devastating muscle-wasting diseases. Created in 1958 by a group of patients and their families, and recognised as being of public utility in 1976, it has set itself two missions: curing neuromuscular diseases and reducing the disabilities they cause.

www.afm-france.org

Genzyme

Genzyme has pioneered the development and delivery of transformative therapies for over 30 years. Founded in 1981 in Boston, Massachusetts, Genzyme evolved from a tiny start-up with just a handful of employees to one of the world’s leading biotech companies. Acquired by Sanofi in 2011, Genzyme now benefits from the reach and resources of one of the world’s largest pharmaceutical companies, with a shared commitment to improving the lives of patients.

Genzyme has long been known for our expertise in the class of rare genetic diseases known as lysosomal storage disorders (LSDs). LSDs remain the heart of our company today, but we have also expanded – through both in-house development and strategic acquisitions and partnerships – to other disease areas such as thyroid cancer and multiple sclerosis.

Genosafe

GenoSafe® is a full contract research and consulting organization which specializes in evaluating the quality, safety and efficacy of innovative biological products.

We meet our clients’ specific needs by performing custom studies in strict compliance with regulatory requirements.

GenoSafe is a Good Laboratory Practices (GLP) compliant facility .

GenoSafe is eligible for the CIR (research tax credit). Our clients may include the R&D Services performed at GenoSafe in their own research tax credit calculation.

REGENX BIOSCIENCE

REGENXBIO is a leading biotechnology company focused on the development, commercialization and licensing of recombinant adeno-associated virus (AAV) gene therapy. Our NAV® Technology Platform, a proprietary AAV gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. Our mission is to transform the lives of patients suffering from severe diseases with significant unmet medical needs by developing and commercializing in vivo gene therapy products based on our NAV Technology Platform. We seek to accomplish this mission through a combination of our internal development efforts and the efforts of our third-party licensees.

Eufets

EUFETS (Germany) is a Contract Development and Manufacturing Organisation specialised in the industrialisation of cell and gene therapy products (viral vectors, cell products and in vitro transcribed mRNA). Based on extensive expertise in molecular biology, virology and cell biology as well as an understanding of the regulatory prerequisites, our GMP experts support you to develop and manufacture your products in a safe and cost-efficient way. We offer a complete service spectrum from process and assay development through clinical trial medication to in-market supply in our state-of-the-art GMP facility.

Come and visit us at booth 14.

Finnvectors

FinVector is a world leader in the research and development of Viral-Based Gene Therapy products, with state-of-the-art facilities and a highly experienced scientific team working in the gene therapy market. We deliver a tailored service to meet and exceed our clients’ needs, and use our scientific expertise and industry knowledge to help clients take viral-based products from the pre-clinical phase, through clinical trials and to the market.

cellular dynamics

Cellular Dynamics International, a FUJIFILM company, develops and manufactures biologically relevant human cells derived from induced pluripotent stem (iPS) cells. Our iCell® and donor-specific MyCell® Products are highly pure, highly reproducible, and available in industrial quantity to enable drug discovery, toxicity testing, stem cell banking, and cell therapy development.

Cobra Bio

About Cobra Biologics: Cobra Biologics is a leading international clinical and commercial manufacturer of biologics and pharmaceuticals with three GMP approved facilities. We offer a broad range of integrated and stand-alone development services, stretching from cell line development through to the commercial supply of investigational medicinal product. We take pride in manufacturing excellence and being a trusted provider, delivering what we promise and helping our customers to develop drugs for the benefit of patients. Cobra Biologics provides manufacturing solutions to the biologics and pharmaceutical industry covering antibodies, recombinant proteins, viruses, DNA, cellular therapeutics and small molecules.

Orchard therapeutics

Orchard Therapeutics is a clinical-stage biotechnology company with operations in London and the United States and dedicated to bringing transformative gene therapies to patients with serious and life-threatening orphan diseases. 

Intellia

Intellia Therapeutics is a leading gene editing company, focused on the development of proprietary, potentially curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course. Our combination of deep scientific expertise and clinical development experience, along with our leading intellectual property portfolio, puts us in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Intellia was named as one of the top 10 biotech start-ups by Nature Biotechnology. In September 2015, Intellia was named a “Fierce 15” biotech company by FierceBiotech

Pfizer

Pfizer and Rare Diseases
Rare diseases are among the most serious of all illnesses and impact millions of patients worldwide, representing an opportunity to apply our knowledge and expertise to help make a significant impact in addressing unmet medical needs. The Pfizer focus on rare diseases builds on more than two decades of experience, a dedicated research unit focusing on rare diseases, and a global portfolio of more than 20 medicines approved worldwide that treat rare diseases in the areas of hematology, neuroscience, inherited metabolic disorders, pulmonology, and oncology.

Pfizer Inc: Working together for a healthier world®
At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products. Our global portfolio includes medicines and vaccines as well as many of the world's best-known consumer health care products. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, Pfizer has worked to make a difference for all who rely on us. For more information, in addition, to learn more, follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

Brammer Bio

Brammer Bio is a contract development and manufacturing organization (CDMO) serving companies seeking to develop and commercialize cell and gene therapies. With an experienced management team and exceptional scientific expertise and proven manufacturing capabilities we offer the knowledge and resources necessary to help you deliver innovative cell and gene-based therapies.

Myocure

Development of an innovative gene therapy platform to cure rare hereditary muscle disorders (#667751; ~€6 million; 4 years), is an EU-wide-H2020 research project funded by the European Commission. MYOCURE aimed to develop a novel, innovative and clinically-translatable one-treatment platform for patients suffering from rare inherited muscle diseases, specifically focusing on myotubular myopathy (MTM) and glycogen storage disorder (GSD) type II. The objectives of MYOCURE are: (1) To boost gene transfer and expression using muscle-specific vectors, (2) To minimize undesirable immune reactions and to validate the efficacy and safety of the innovative vector platform technology in preclinical MTM and GSD II animal models, (3) To develop a scalable GMP-like manufacturing process for subsequent GLP toxicology studies, (4) To apply for an orphan drug designation for the innovative advanced therapy medicinal product (ATMP), (5) To consolidate a Phase I gene therapy clinical trial in patients suffering from MTM and GSD 

Biomarin

With six products on the market and a fully-integrated multinational organization in place, BioMarin is providing innovative therapeutics to patients with serious unmet medical needs. The company is also currently conducting a clinical trial of an AAV-based potential gene therapy for hemophilia A.

PEI

The Paul-Ehrlich-Institut (PEI), Federal Institute for Vaccines and Biomedicines, is a world-renowned scientific institute devoted to improving public health by ensuring the safety and efficacy of vaccines and biomedicines both in Germany and throughout the world. PEI carries out its mission through scientific research and medicinal product regulation. Internationally competitive research is carried out in the fields of virology, microbiology, allergology, immunology, hematology and cell and gene therapy.

Molecular Therapy

Molecular Therapy, the official flagship journal of the American Society of Gene & Cell Therapy, is the leading international journal for basic, translation and clinical research in the areas of gene and cell therapy. The journal covers gene transfer, vector development and design, stem cell manipulation, development of gene-, peptide-, protein-, oligonucleotide-, and cell-based therapeutics to correct genetic and acquired diseases, vaccine development, pre-clinical target validation, safety/efficacy studies, and clinical trials. The Molecular Therapy  family of journals also includes the open-access online sibling journals MT-Nucleic AcidsMT-Oncoloytics, and MT-Methods and Clinical Development.  Publishing important peer-reviewed research and cutting-edge reviews and commentaries, the journals continue to attract the best material in the field.

Shire

Founded more than 30 years ago, we have long believed we have a unique opportunity to champion underserved patient communities. Today, as the global leader in rare diseases, we are determined to build on this position as we go forward.

Speed matters, especially to the patients who are waiting for treatments and diagnosis. As we grow, we want to retain the spirit of a small biotech with the benefits of both scale and agility. We do that through flat and rapid decision-making and operational excellence with the interplay among our key strategic centers in Boston, Dublin and Zug. Through a fast-paced, entrepreneurial and international culture, we give people freedom and opportunity to excel while also setting a high bar for ethics and responsibility.

Bronze

Apceth

apceth Biopharma is a pioneering biopharmaceutical company with an innovative pipeline of cell-based gene therapeutics for the treatment of GvHD, solid cancer and major chronic diseases (lung diseases, metabolic and autoimmune diseases). The company's proprietary platform technology is based on state-of-the-art genetic engineering of mesenchymal stem cells (MSC). In parallel, apceth Biopharma is also a successful Contract Development and Manufacturing Organization (CDMO) for complex cell and gene therapy products with a high international reputation. Based in Munich (Germany) in the heart of Europe, apceth Biopharma provides its proven expertise and state-of-the-art GMP facilities to clients from around the world.

Kite Pharma

Kite is a biopharmaceutical company engaged in the development of innovative cancer immunotherapies with a goal of providing rapid, long-term durable response and eliminating the burden of chronic care. The company is focused on chimeric antigen receptor (CAR) and T cell receptor (TCR) engineered cell therapies designed to empower the immune system's ability to recognize and kill tumors. Kite is based in Santa Monica, CA.

BioNTech

BioNTech is Europe’s largest privately held biopharmaceutical company pioneering the development of individualized therapies for cancer and other diseases. The company combines all building blocks for individualized immunotherapy under one roof – from diagnostics and drug development to manufacturing. Its cutting-edge technologies range from individualized mRNA-based medicines through innovative chimeric antigen receptors and T-cell receptor-based products to novel checkpoint immunomodulators. BioNTech’s approach is validated by five top-tier corporate partnerships with Genentech, Genmab, Eli Lilly and Company, Sanofi and Bayer Animal Health. Founded in 2008, BioNTech’s shareholders include the MIG Fonds, Salvia and the Strüngmann Family Office, with the Strüngmann Family Office as the majority shareholder.

Accelero

Your Contract Service Provider for Advanced Therapy Medicinal Products (ATMP) in Berlin, Germany.

YOUR ANALYTICAL PARTNER FOR:

  • Non-clinical drug development.
  • Clinical drug development.
  • Independent contract research.

ACCELERO IS ...

  • Good Laboratory Practice (GLP) certified.
  • Compliant to ICH GCP regulations.
  • Approved as BSL-2 laboratories for genetic engineering, microbiology and cell culture.
  • Registered for the import of animal products from non-EU origins.

ACCELERO PROVIDES …

  • Validated qPCR assays for regulatory biodistribution studies.
  • ELISA.
  • Flow cytometry.
  • Microarray biomarker screening.
  • Cell culture and microbiology.
  • Immunology assays.
  • Bioanalytical consulting.
  • GLP training program.

ACCELERO APPLIES FOR …

  • Pharmacokinetic and toxicokinetic characterization.
  • ATMP biodistribution.
  • Impurity tests.
  • Formulation analysis.
  • Pharmacodynamics.
  • Biomarker research.
  • Cell-based assays.
  • Cloning and gene expression analysis.

Supporter

LOEWE CGT

LOEWE Center for Cell and Gene Therapy Frankfurt

The Center for Cell and Gene Therapy Frankfurt has been established in 2011 to foster the development of advanced therapy medicinal products in the areas of hematology, immunology and cardiology. The center, which is funded by the “Hessisches Ministerium für Wissenschaft und Kunst”, brings together research groups from the University Medical Center, the Georg-Speyer-Haus, the Paul-Ehrlich-Institute, the Institute of Transfusion Medicine and the Max-Planck Institute for Heart and Lung Function generating a center of excellence in regenerative medicine. The aim of the center is to bring innovative therapeutic concepts into clinical application.

Six different research areas are essential for the LOEWE-CGT:

1. regenerative medicine

2. stem cell transplantation and immunotherapy

3. stem cell therapy

4. cardiovascular disease

5. signal transduction and stem cell biology

6. risk analysis

Springer Healthcare

New Horizons in the management of ADA-SCID is a free, independent, online resource dedicated to providing cutting-edge information to Immunologists, Gene and Cell Therapy Clinicians, Transplant Specialists, Paediatricians and Haematologists, faced with Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) patients. This educational resource led by Dr Andrew Gennery, Professor Bobby Gaspar and Dr Robbert Bredius, provides you with an expert-led collection of up-to-date information and practical insights in one location to support you in diagnosing and treating patients with ADA-SCID. Visit ada-scid.online for summaries of the latest research, free access to full-text articles, interviews with leading experts, interactive patient case studies and expert-led webinars. New Horizons in the Management of ADA-SCID is produced by Springer Healthcare IME.