WHAT IS THE WORLD ADVANCED THERAPIES & REGENERATIVE MEDICINE CONGRESS?
Europe’s most important advanced therapies conference and exhibition with 1000+ attendees
and 300+ speakers.
The agenda covers gene therapy, gene edited cell therapies, stem cells & regenerative medicine. The event covers each stage of development of cell, gene and immunotherapy - from bioprocessing through clinical translation to reimbursement, market access and delivery to patient.
15–17 May 2019 - Conference Days
16-17 May 2019 - Exhibition Days
Business Design Centre, London, UK
Research. Develop. Collaborate. Commercialise.
Join the Congress and Expo on 15-17 May in London for the most content packed three days that the World Advanced Therapy & Regenerative Medicine Congress has to offer yet.
Visit the website HERE to book your place, early bird offer if you book before the 26 April you can save up to £270!
If you think you know the World Advanced Therapy & Regenerative Medicine Congress because you’ve attended before, think again. With all that’s going on this year, this is not an event to miss.
For more information
World Advanced Therapies & Regenerative Medicine Congress
+44 207 092 1150
Brussels, 23rd April 2019 – The 9th World Primary Immunodeficiency (PI) Week campaign starts today, with one key message: let’s deliver care with and for patients with primary immunodeficiency worldwide.
Spotlight on patient-centricity
There are over 320 different types of primary immunodeficiencies, affecting over 6 million people worldwide. The types are all different, meaning that each individual requires a personalised approach, focused on his/her specific needs. World PI Week, 22-29 April, is an opportunity to join a global movement calling for patient-centred care for people living with primary immunodeficiency and their families around the world.
Patient centricity means not working around but with patients: patients and their families are partners of healthcare providers in the decisions related to their own care and treatment plans.
Patient centricity requires re-thinking and re-organising services so they are truly oriented to deliver the best value to their end-users, the patients. One example of this is the systematic implementation of new born screening for severe forms of primary immunodeficiency, which ensures that patients can be diagnosed at an early stage.
Patient centricity also means working in a holistic, inclusive way with the multiple actors who play a role in care delivery, from patients and doctors, to allied healthcare professionals, biologists and researchers.
Spreading the word across the globe to deliver change
From today onwards, people on all continents are taking part in the World PI Week by organising events, conferences, family days, TV/radio interviews and many other activities to raise awareness and help bring about change.
Get involved to show support of the primary immunodeficiency community! Join the many individuals living with the disease, their families, healthcare professionals, scientific experts, companies, policy-makers and researchers across the globe who are advocating for a political, societal and healthcare shift towards patient-centricity. This will bring the best outcomes for primary immunodeficiency patients!
Capitalising on its new branding and vision and supported by its active network, the World PI Week 2019 brings positive momentum for action.
Together, bringing about change for primary immunodeficiency patients worldwide!
About primary immunodeficiencies
Primary immunodeficiencies are rare diseases which occur when a person’s immune system is absent or does not function properly. When a defect in the immune system is inherited (carried through the genes), it is called primary immunodeficiency. There are over 320 forms of Primary Immunodeficiency (PI or PID), ranging widely in severity.
Primary Immunodeficiency often presents in the form of “common” infections, sometimes leading physicians to treat the infections while missing the underlying cause, allowing the infections to reoccur, and leaving the patient vulnerable to vital organ damage, physical disability, and even death.
For more information, please visit www.worldpiweek.org, follow us on Twitter @WorldPIWeek.
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Bénédicte Faure, campaign manager: email@example.com
EspeRare enters into partnership with Dermelix Biotherapeutics to develop DMX-101, an in utero treatment for X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)
Geneva, Switzerland – 2 April, 2019 – EspeRare, a not-for-profit organization dedicated to accelerating the development of rare diseases treatments, today announced that it has entered into an agreement with Dermelix Biotherapeutics for the co-development of its lead programme, DMX-101. DMX-101 is a novel in utero protein replacement therapy for the treatment of X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED), a rare pediatric genetic disease.
Under the terms of the agreement, EspeRare will sponsor the development of DMX-101 (previously ER-004) in Europe, where it was accepted under the EMA’s PRIME (Priority Medicines) scheme and benefits from Orphan Drug Designation. Dermelix will sponsor the development of DMX-101 outside of Europe and will be responsible for its commercialization worldwide.
XLHED is a rare genetic disorder affecting ectodermal structures including sweat glands, respiratory glands, skin, hair and teeth. Clinical manifestations of XLHED are severe and can include life-threatening episodes of hyperthermia, heat intolerance, and an increased risk of serious respiratory tract infections. There are currently no approved therapies for treatment of XLHED and the current standard of care is only palliative.
DMX-101 is a protein replacement therapy designed as a substitute for endogenous EDA, a protein missing in XLHED. It is administered during late foetal development through a single-course treatment delivered into the amniotic fluid. This approach has already demonstrated significant benefits in a prenatal study, the results of which were recently published in the New England Journal of Medicine1 and featured in Nature Medicine’s 2018 Research Highlights2.
In the second half of 2019, EspeRare and Dermelix anticipate the start of patient enrollment into a pivotal study for DMX-101, first in Europe and then in the US, with the aim of moving the treatment towards market approval. In the US, DMX-101 benefits from Orphan Drug Designation and Fast Track Designation by the FDA.
Dr. Nick France, Chief Medical Officer of Dermelix, commented: “It is both tremendously exciting and humbling to be able to participate in such a groundbreaking program. The ability to correct a severe disease before birth represents a huge step forward in therapeutic paradigms. We look forward to a productive collaboration with EspeRare and working hard for families with XLHED.”
In line with EspeRare’s model and in order to reflect EspeRare and Dermelix’ common values and patient-centric approach, the agreement also includes an Ethics and Social Responsibility Charter under which both partners have committed to fully and transparently engage the patient community. Through a Patient Advisory Council, the partners will streamline information transfer in order to provide valuable inputs for the development of DMX-101.
XLHED is a severe, chronically debilitating and life-threatening rare disease affecting approximately 4/100,000 live male births every year. XLHED is caused by genetic mutations in the EDA gene, a gene that encodes for an important ectodermal developmental protein, EDA. The absence of functional EDA results in abnormal development of the skin, sweat glands, sebaceous glands, hair, oral cavity, and respiratory mucosal glands resulting in serious life-threatening clinical manifestations from birth including hyperthermia, craniofacial anomalies and recurrent respiratory infections that impair quality of life in patients and their families.
For more information, please visit https://bit.ly/2KbMqGa
EspeRare is a Swiss not-for-profit organization that is committed to improve the lives of children with life-threatening rare diseases. EspeRare addresses the unmet medical needs of these children by uncovering the potential of existing treatments. EspeRare’s innovative model combines pharmaceutical know-how with philanthropic, public and private investments to develop and bring to life these discontinued therapies. With its unique patient-centered approach to drug development, EspeRare engages the patient community at each step of the process, with the intent of giving children and their families fair access to these therapies and a new hope for the future. For more information, please visit https://esperare.org/en/node/13
DMX-101 is a fully humanized EDA molecule consisting of the human IgG1 Fc sequence linked to the human EDA TNF binding domain. Preclinically, DMX-101 has been shown to bind to the receptor EDAR resulting in activation of the NFϰB signaling pathway, which triggers the transcription of genes involved in the normal development of multiple tissue types. DMX-101 is the first and only treatment specifically targeting XLHED. Administered during the third trimester of pregnancy, it has the potential to become a “single course” treatment, significantly improving symptoms of the disease throughout patients’ lives. This approach has already demonstrated significant potential in in a case series of three patients treated in utero with DMX-101 during the third trimester of pregnancy. The treatment normalized sweat gland function and associated thermoregulation, and improvement in dentition and respiratory function were observed. These results were recently published in the New England Journal of Medicine1 and featured in Nature Medicine’s 2018 Research Highlights2.
For more information, please visit https://esperare.org/en/dmx-101
About Dermelix Biotherapeutics
Dermelix is a privately-held, clinical-stage biopharmaceutical company focused on the development of innovative therapies for rare and debilitating dermatologic conditions with high unmet medical need. For more information, please visit https://www.dermelix.com
For more information please contact:
For English-speaking and International Media:
Instinctif Partners for EspeRare
Sue Charles / Dr Christelle Kerouedan / Genevieve Wilson
T: +44 (0) 20 7457 2020
Rare Revolution Editor