News

02Jul

EspeRare Returns to NFED's Annual Conference to Present Edelife Trial

NFED, EDELIFE clinical trial, rare disease, XLHED

EspeRare is delighted to announce our return to the National Foundation for Ectodermal Dysplasias (NFED) Annual Family Conference, taking place from July 7-9, 2024, at Grand Hyatt, Washington, D.C. We will be presenting our EDELIFE trial for X-linked Hypohidrotic Ectodermal Dysplasia (XLHED) Edelife | XLHED Clinical Trial I For Pregnant Women (edelifeclinicaltrial.com), including the opening of a new clinical site in the US, in Los Angeles. The Principal Investigators in the US, Prof Dorothy Grange and Prof Ophir Klein, will also be present and available for discussion.

This year, the NFED is having a Day on the Capitol Hill, on Monday, July 8, to advocate for and make a difference for those living with ectodermal dysplasia and other congenital anomalies. Pierre Fabre and EspeRare are proud to be standing with the NFED.

For more details on the event, please visit the NFED Stand Together 2024 event page.

NFED 2024

19Jun

EspeRare to be featured at the Fongit’s Life Sciences Event at the Campus Biotech

On June 25th 2024 starting at 9AM, EspeRare, a pioneering nonprofit biotech organization, will be featured at the Fongit Café Croissant event at Campus Biotech. During this session, EspeRare's Executive Director and Co-Founder, Caroline Kant will present the organization's latest developments and answer questions from attendees.

https://www.fongit.ch/event-details-registration/cafe-croissant-at-campus-biotech-1-2

About Fongit: For over three decades, Fongit has been de-risking the entrepreneurial journey and supporting entrepreneurs with the expertise, resources, and financing they need to transform innovative ideas into sustainable companies. As a private, non-profit foundation supported by the State of Geneva, Fongit’s mission is to convert innovation into social and economic value in the Geneva region.

About the Event: This monthly event gathers life sciences entrepreneurs from the community, providing a platform to discover the latest happenings in the Geneva ecosystem. Attendees will have the opportunity to hear directly from entrepreneurs, learn about their journeys, and expand their professional networks over coffee and croissants.

13Jun

EspeRare participates in the 21st World Congress in Fetal Medicine, Lisbon, PT

EspeRare is participating in the 21st World Congress in Fetal Medicine, happening in Lisbon, Portugal, from June 23rd to June 27th, 2024.

We are excited to showcase our groundbreaking prenatal Edelife clinical study and to delve into the latest advancements in the field of prenatal medicine. This congress provides an exceptional platform to exchange insights and collaborate with leading experts dedicated to enhancing fetal health and care.

If you are attending and would like to exchange ideas or meet with the EspeRare team, please connect with Agnes Jaulent and Sebastien Mazzuri, who will be on-site at the congress. For more information about the congress and to register, please visit the following link: https://fetalmedicine.org/courses-n-congress/fmf-world-congress

EspeRare Foundation and the Pierre Fabre Group run a pivotal clinical trial, Edelife, aimed at confirming the safety and efficacy of ER-004, a prenatal treatment for XLHED (X-linked Hypohidrotic Ectodermal Dysplasia), a rare and debilitating congenital disease. Upon positive results, the study could lead to the first approved in-utero treatment for XLHED by 2026.

For more information on the Edelife clinical trial please follow the link: XLHED Ectodermal Dysplasia ER004 Clinical Study (edelifeclinicaltrial.com)

Fetal Medicine

12Jun

EspeRare being featured on 'Objectif croissance' program, France

Our R&D Director, Florence Porte-Thomé was recently featured in an engaging interview on the 'Objectif croissance' program, where she presented EspeRare's "venture philanthropy" model and cutting-edge innovations in drug development for rare diseases. The discussion was vibrant and inspiring, highlighting EspeRare's commitment to transforming lives through science.

More insights and the full interview coming soon.

BFM

22May

EspeRare's 2023 Annual Report is available

Foundation, Esperare, Annual report

EspeRare's Annual Report for 2023 has just been published.

The report provides a comprehensive overview of Foundation's financial performance, strategic initiatives, and key achievements over the past year.

Click here to read our new Annual Report.

29Feb

Rare Disease Day 2024

Today is a very special day, Rare Disease Day! A day that gives us the opportunity to shine a spotlight on patients and families living with rare conditions. It is also a special moment in time to raise awareness about research study conducted for boys with XLHED, a rare genetic disease that can affect the sweat glands, respiration, skin, hair, teeth, and may significantly impacts quality of life. A clinical trial, Edelife is underway to evaluate a potential treatment for the most common form of ectodermal dysplasia (XLHED).

Pregnant women in any country who are XLHED carriers and pregnant with a boy may be able to join.

Recruitment is ongoing !

https://edelifeclinicaltrial.com/

22Feb

Celebrating EURORDIS Company Award for Patient Engagement 2024

On February 20, 2024, the EspeRare Foundation and Pierre Fabre Laboratories received a prestigious EURORDIS Company Award for Patient Engagement. It was an extraordinary evening marking a significant milestone in our journey and an enriching collaboration with patient groups.

We feel immensely honored to be recognized for the remarkable efforts we make with and for those living with a rare disease. It truly exemplifies the spirit of collaboration and perseverance that defines the rare disease community. Working hand-in-hand with the Ectodermal Dysplasias representatives we learned how to better communicate with the families, understand their needs, preferences and challenges. We were able to echo the patient voice into our clinical study making it truly patient-centric, inclusive, trustworthy and accessible to families that consider participating. We are hopeful that our groundbreaking prenatal treatment aiming to correct symptoms of X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED), a rare genetic disease, will truly address their needs and become accessible to patients.

This remarkable evening, filled with emotions and amazing people, reinforces our hope and inspiration to continue to strive for excellence, innovation and pushing the boundaries of responsible drug development for the rare disease community.

The awards ceremony serves to acknowledge the dedication and innovation demonstrated by individuals and organizations committed to advancing the cause of rare diseases. From patient advocates to policymakers, scientists to companies, the event shines a spotlight on those who tirelessly work towards improving the lives of those affected by rare diseases.

To find out more on the Black Pearl Award please follow the link: Black Pearl Awards Homepage - EURORDIS

30Jan

EspeRare Foundation and Pierre Fabre Laboratories to be honored at EURORDIS Black Pearl Award Ceremony for Patient Engagement 2024

Pierre Fabre Laboratories and the EspeRare Foundation will receive the “EURORDIS Company Award for Patient Engagement 2024”. The award ceremony, a part of the EURORDIS Black Pearl Awards scheduled for February 20^th2024 will take place in Brussels (Belgium), recognizing the exemplary efforts and long-standing collaboration they have established with the Patient groups in X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED).

EspeRare and Pierre Fabre, combined their complementary expertise in 2020 to jointly develop a groundbreaking prenatal therapy for XLHED patients, with the hope of delivering the first treatment to address this genetic condition before birth. Their shared commitment, values, and principles not only drive the development of this pioneering treatment but also empower the rare disease community to actively participate and contribute their insights at every stage of the drug development process. EspeRare and Pierre Fabre are genuinely honored and grateful to receive acknowledgement for their collaborative, patient-centric approach.

The inauguration day is even more remarkable as it takes place on the Ectodermal Dysplasias Awareness Day, a day that puts orphan disease community together to advocate about these rare conditions and the work being done. This makes Pierre Fabre and EspeRare extremely privileged to have a meaningful involvement with the patient community and collaboratively develop this potentially life-changing therapy being currently tested in the EDELIFE clinical trial.

For more information on the Eurordis Black Pearl Award please follow the link: Black Pearl Awards - Awardees - EURORDIS

08Nov

EspeRare being featured during Global Entrepreneurship Week

November 13, 2023, EspeRare is participating in an illuminating fireside chat, hosted by >>venture>>, focusing on the significant role of nonprofits and impact entrepreneurship ventures in driving innovation. The event will feature an enriching discussion between Caroline Kant, the esteemed founder of EspeRare, and Lea Firmin, CEO of >>venture>>.

Be part of this insightful conversation and reserve your free ticket at: Innovation in Nonprofits & Social Entrepreneurship Tickets, Mon, Nov 13, 2023 at 1:45 PM | Eventbrite

12Oct

HUG to Host Innovation Day on 19th October with a Keynote from EspeRare’s co-founder, Caroline Kant

Esperare, News, XLHED

EspeRare, a pioneer in prenatal treatment advancements, announces that its co-founder and executive director, Caroline Kant, will be featured as a keynote speaker at the Geneva University Hospitals (HUG) Innovation Day on October 19, 2023.

The spotlight of Caroline’s keynote will be "EspeRare: a collaborative model at the forefront of prenatal treatment." Attendees will be given an insightful glimpse into the groundbreaking work EspeRare is undertaking, particularly surrounding ER-004 in X-Linked ectodermal dysplasia. This program, rescued and championed by EspeRare, is poised to become the first-ever treatment that addresses a genetic disease before birth, setting a groundbreaking precedent in medical innovation and offering hope for countless genetic conditions with debilitating perinatal symptoms.

About HUG Innovation Day

The HUG Innovation Day, now in its 17th year, is a platform that showcases innovative health projects. As an established event, it gathers innovators, allowing them to network, exchange experiences, and present transformative ideas within the healthcare sphere. Awards, including the Innovation Award, a Jury’s Favorite, and audience-driven prize, will highlight the day’s presentations. A startup award will also aim to support the ambitions of emerging healthcare entrepreneurs.

For those interested in attending or learning more about the HUG Innovation Day 2023, additional details can be found at: https://www.hug.ch/centre-linnovation/journee-linnovation-2023.

For more about EspeRare and ER004 in XLHED, visit ER-004 Programme in X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED) | EspeRare Foundation

11Oct

Our 10th Anniversary Highlights Video Now Available!

We are delighted to announce the release of our 10th Anniversary Highlights Video, which encapsulates the essence of our remarkable journey over the past decade, as well as our ambitious vision for the future. As we take this opportunity to reflect on our journey, we acknowledge the significant contributions of our public and philanthropic donors, scientific community, and the unwavering support of our valued rare disease ecosystem. We are confident that through continued collaboration, we can achieve even greater progress in paving the path towards a more equitable future.

Click on the photo to view the video and be part of the celebration:

22Jun

EspeRare celebrated a Decade of Daring to Care!

On Thursday, June 8^th 2023 EspeRare had an immense pleasure of celebrating its 10^th anniversary at Campus Biotech in Geneva, CH. It was an extraordinary evening, made even more special by the presence of Foundation’s dedicated and supportive guests. Their energy and enthusiasm truly illuminated the occasion!

The official ceremony featured the heartfelt patient stories and uplifting speeches given by esteemed medical figures, all centered around the principles of embracing research, patient-centricity and collaboration. The festivities finished with the cocktail dinner reception in the entourage of the immersive photography exhibition, Days of Rare, created by Ceridwen Hughes and SbD Creative.

It was a truly remarkable evening filled with emotions, hope and inspiration to continue striving for excellence, innovation and pushing the boundaries of responsible drug development for the rare disease community.

The next chapter has begun, and EspeRare will continue to make a lasting impact and create a brighter future for rare disease patients worldwide.

Merci

23Feb

EspeRare is present at the Rare Disease Day at NIH 2023

EspeRare and its partner Pierre Fabre Group will participate in the Rare Disease Day at NIH on 28^th February 2023, presenting their ground-breaking clinical study for families with XLHED. The virtual booth will give the possibility to attendees from various parts of the world to connect and find out more about the new treatment paradigm and engage with the speakers.

National Institutes of Health (NIH), through this event aims to raise awareness about rare diseases, the people they affect, and NIH collaborations that address scientific challenges and advance research for new treatments. The event is free and open to the public. Registration is required prior to attending: https://hopin.com/events/nih-rare-disease-day-2023/registration

02Feb

Pierre Fabre and EspeRare move forward a pioneering in-utero therapy for XLHED patients

When French pharma company, Laboratoires Pierre Fabre and Swiss not-for-profit organization, EspeRare, joined forces by the end of 2020, their goal was to develop a life-changing treatment for a debilitating rare disease, X-Linked Hypohidrotic Ectodermal Dysplasia, which affects approximately 1 in 25,000 live male births every year.

“Currently, there are no therapeutic options for XLHED. Only the symptoms of the disease can be alleviated” – as Caroline Kant, CEO and co-founder of EspeRare and Caroline Miklaszewski, Project Director at Pierre Fabre, jointly stated.

There is no doubt that the drug development in rare diseases is highly challenging but when two patient-centric organizations combine complementary expertise, the ground-breaking research and a novel therapeutic approach can see a daylight. ER-004, a pioneering in-utero protein replacement therapy, is currently being investigated in the EDELIFE clinical trial and if approved, it will become the first therapy to address a genetic condition before birth.

To read the full article, please follow the link: Discovery & Development (themedicinemaker.com)

10Jan

Repurposing drugs in a novel way and a call for proposals for advocacy organizations

In the context of therapy development webinar series launched in November 2022 by Advocacy Leaders: Genetic Alliance, EspeRare Foundation and Luna, EspeRare will take a lead at the third webinar taking place on 18^th January 2023. The Foundation will present how shelved drugs can accelerate the development of life-changing therapies and lay out the practical examples based on its programmes in X-linked Hypohidrotic Ectodermal Dysplasia and Duchenne. During this webinar, a call for proposals will be launched and EspeRare will encourage its audience to engage and potentially co-develop through a partnership a new therapeutic opportunity for its community addressing high unmet medical needs in rare diseases. Finally, Luna will run two final webinars to demonstrate major features of its Community Driven Innovation methodology (15th Feb) and its innovative registry and studies platform (1st Mar).

Here is the link for the survey mentioned: Therapy Development - Call for Proposals. Deadline for submissions is February 1^st 2023.

Webinars are open to public. For more information on the series of webinars and registration please visit the link.

03Jan

NeoCare featured in the latest issue of Le Micromécanicien

Neocare, CHD, Foundation

Miniaturization and precision at the service of life.

The development of medical devices requires not only the knowledge of medical experts, but also engineers and innovative technologies. The journal Le Micromécanicien, in its December edition, published an article on NeoCare - an innovative medical device aimed at protecting the heart and lungs of newborns with heart defects.

A great opportunity for a medtech to finalize the development, production and commercialization of NeoCare for congenital heart defects and to extend this technology to many other indications for which the control and regulation of vessel pressure is necessary, for example liver transplantation or major liver resection.

To read the full article go to pages 30-31 on the following link: https://lemicromecanicien.ch/

About Micromécanicien:

Le Micromécanicien is a specialized, technical and industrial magazine dedicated to microtechnology. Microtechnology applies to a field that permeates practically the entire industrial sector, particularly in French-speaking Switzerland, with a strong presence in the Jura Arc.

14Dec

Appointment of Bertrand Kiefer, Medical and Bioethics expert, as new board member

EspeRare is delighted to announce the appointment of Bertrand Kiefer, PhD in medicine and bioethics as a new member of the Foundation Board. This distinguished member of the Geneva and Swiss scientific community brings a wealth of expertise in medicine, bioethics, theology and journalism, all of which will be of considerable value to the governance of EspeRare.

“Bertrand Kiefer affords us the privilege of joining the Foundation on the eve of its tenth anniversary. His unique experience and fierce commitment to the medical and ethical fields is an invaluable asset in guiding EspeRare through the next stage of its development” commented Caroline Kant, co-founder, and executive director.

After receiving his medical degree in infectious diseases from the University of Geneva, Dr. Kiefer studied theology and philosophy of science at the University of Fribourg and then in Rome, before engaging in bioethics research. He is also a trained journalist and is the author of over 600 articles, columns and editorials. He is also editor-in-chief of the Swiss Medical Journal.

In addition to being a member of the board of various not- for -profit associations and committees in Switzerland, Bertrand Kiefer was a member of the Swiss National Commission for Ethics in the Human Domain for 12 years.

Today, it is a great privilege to welcome Bertrand Kiefer to the Board of Foundation EspeRare. There is no doubt that his contribution, along with the contribution of the other dedicated members of the Foundation Board, will be instrumental to the success of the Foundation in the years to come.

28Nov

Launching a Therapy Development Webinar Series & a call for proposal for advocacy organizations

As advocacy leaders, Genetic Alliance, EspeRare and Luna are joining forces to support the active participation of advocacy community in therapeutic development. To this end, over the next 4 months, Genetic Alliance will moderate a Webinar Series about the drug development process. The first two webinars (29^th Nov & 14^th Dec) will focus on presenting the NETS, a dynamic, interactive map that intends to provide a realistic view of drug development, and its associated resources helpful for understanding and executing that process. On January 18^th, EspeRare will present how shelved drugs can accelerate the development of life-changing therapies and lay out the practical examples based on its programmes. During this webinar, a call for proposal will be launched and EspeRare will commit its expertise and support to an advocacy organization to co-develop a new therapeutic program addressing high unmet medical needs in rare diseases. Finally, Luna will run two final webinars demonstrate major features of its Community Driven Innovation methodology (15^th Feb) and its innovative registry and studies platform (1^stMar).

For more information on the series of webinars and registration please visit the link.

05Oct

Hope for Rare – Inspirational interview with Caroline Kant

EspeRare’s co-founder and Executive Director, Caroline Kant, was honoured to share her story within the framework of rare diseases which was feathered in the 3^rd edition of Rarity Life. Starting with the pharma experience, she then moves on to describing the opportunity for establishing a new collaborative business model focused on patient inclusion and the accessibility to treatments.

To read the full article go to page 46 on the following link https://issuu.com/raritylife/docs/rarity_life_3

Rarity Life is a brand new online publication that offers those affected by rare diseases, disability and cancer the opportunity to create content that is truly inclusive. Apart from art, culture and fashion, the magazine includes life stories and inspirational people doing remarkable work within their community.

To subscribe to Rarity Life go to: www.samebutdifferentcic.org.uk/raritylife

26Sep

EspeRare, Pierre Fabre Group and EDS are present at DrugRepo22 in London, UK

EspeRare, their partner Pierre Fabre Group and the UK patient association will be presenting at the Drug Repurposing for Rare Diseases Conference in London on October 10^th 2022 on how they are joining forces to reposition and develop a landmark treatment in XLHED.

EspeRare will also present its pioneering new collaborative model centered on patients and integrating the ethical commitment in terms of the accessibility to treatments for rare disease community.

This annual conference, live and in-person since 2020 highlights the role that drug repurposing can play in lowering the cost and accelerating the development of rare disease treatments.

For more information on the DrugRepo 2022, please follow the link.

Advancing treatments for rare diseases together