We’re thrilled to partner with BioSensics on our Charcot-Marie-Tooth disease type 4J (CMT4J) natural history study. A big thank you to Ashkan Vaziri and the entire BioSensics team for being outstanding collaborators in this important work. CureCMT4J/Talia Duff Foundation Elpida Therapeutics California Institute for Regenerative Medicine (CIRM) The National Institutes of Health UT Southwestern Medical Center Stanford University Iowa State University #raredisease #CMT4J #OurChildren https://lnkd.in/gn7mrFiT
Elpida Therapeutics
生物技术研究
Los Angeles，California 1,385 位关注者
Accelerating Life Saving Gene Therapies
关于我们
At Elpida Therapeutics our main goal is to deliver Gene Therapies to patients in need. We leverage our years of experience along with our strong partners to enable rapid approval and exponential program growth & adaption!
- 网站
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Elpidatx.com
Elpida Therapeutics的外部链接
- 所属行业
- 生物技术研究
- 规模
- 2-10 人
- 总部
- Los Angeles，California
- 类型
- 非营利机构
地点
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主要
获取路线
US，California，Los Angeles
Elpida Therapeutics员工
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Keith Gottlieb
PhD, Virology/Vaccinology/Gene Therapy
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Laura Hameed
Possibilitarian. Nonprofit Executive, U of MN Regent Emeritus, Board Director, Aspen Institute Rodel Fellow. Accelerating equitable access to…
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Salim Yazji, MD
Executive & Entrepreneur/ Chief Medical Officer
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Rachel Thomas
RN, BSN, CCRC
动态
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We are deeply grateful to California Institute for Regenerative Medicine (CIRM) for their support in raising awareness and providing the funding, guidance, and support needed on our journey to develop treatments for CMT4J and other genetic diseases. -Together, we will turn the impossible into possible!-
In December 2023, CIRM awarded Elpida Therapeutics a $3.9 million grant to support their groundbreaking work on Charcot-Marie-Tooth disease type 4J (CMT4J). CMT4J is an ultra-rare, inherited disorder that severely affects nerves and motor function, with no available treatments or cures. By funding this preclinical study, CIRM is helping to close critical knowledge gaps and accelerate the development of new stem cell and gene therapies. Learn more about CMT4J and what Elpida is doing to advance research forward. #CMT4J #RegenerativeMedicine #CIRM?#RareDiseases #RareDisease #Preclinical #StemCells #GeneTherapies
Story of Hope: Elpida Therapeutics’ Pioneer Study on Rare Nerve Disease CMT4J
https://blog.cirm.ca.gov
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We are truly blessed and grateful to have the incredible support and partnership of Fundación Columbus. Without their backing, Elpida would not exist, and the amazing work we are doing would never have been possible. Together, we will make the impossible possible!
? ?Y nació una nueva alianza! ? En 2023, la Fundación Columbus da un paso más en su misión de transformar vidas al unirse a Elpida Therapeutics, una corporación con propósito social comprometida con las enfermedades raras y la innovación en la terapia génica. ?? ElpidaTx no solo busca la rentabilidad, sino que también trabaja incansablemente para desarrollar terapias génicas que aborden enfermedades raras neurodegenerativas. Su programa líder, Melpida, ya en fase clínica, se enfoca en la enfermedad SPG50, y juntos, aspiramos a expandir este éxito a otros programas similares. Esta colaboración refleja nuestro compromiso conjunto de ofrecer esperanza y soluciones a quienes más lo necesitan. ?Estamos emocionados por lo que nos depara el futuro! ?? #FundaciónColumbus #ElpidaTx #TerapiaGénica #SPG50 #InnovaciónSocial #SaludGlobal #EnfermedadesRaras Elpida Therapeutics CureSPG50 Terry Pirovolakis Viralgen Vector Core
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?? Exciting news! Elpida Therapeutics has enrolled the first patients in its groundbreaking Natural History Study for Charcot-Marie-Tooth Disease Type 4J (CMT4J). This crucial research will help shape future treatments and therapies. Read the full press release for more details: https://lnkd.in/gdugbhgU #CMT4J #GeneTherapy #RareDiseases #MedicalResearch #ElpidaTherapeutic California Institute for Regenerative Medicine (CIRM)
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Elpida TX is dedicated to expediting life-saving gene therapies and enhancing the lives of children affected by rare diseases. This commitment extends beyond our own programs to encompass all afflicted children worldwide. Thanks to remarkable collaborations at UTSW, Viralgen, Boston Children's, SickKids, Columbus Children's Foundation, Foundation Columbus, CureSPG50 & others, we have opted to release our Pre-IND, IND and Clinical documents for our SPG50 program. As a collective, we hope that these documents will aid others on their program journeys??and inspire them to share their documents, fostering an environment of transparency and cooperation for the advancement of rare disease programs globally. https://lnkd.in/gNynw_NS UT Southwestern Medical Center, Viralgen Vector Core, Boston Children's Hospital, The Hospital for Sick Children, Columbus Children's Foundation, Fundación Columbus, CureSPG50
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Thank you California Institute for Regenerative Medicine and BGTC for supporting our programs and funding critical programs for rare diseases. Rare diseases have little to no funding options and we are so thankful for your incredible support!
CIRM is proud to fund and spread awareness of rare diseases this?#RareDiseaseDay. CIRM?funds?rare and prevalent disease research to?advance innovative science and technology to benefit patients with unmet medical needs. In fact, 50% of?our portfolio?is in rare diseases such as: Retinitis Pigmentosa, Sickle cell disease, Huntington’s disease, Duchenne Muscular Dystrophy. CIRM is a?member?of?the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium?(AMP BGTC) to rapidly advance transformative gene therapies for rare diseases, which collectively affect millions of patients. CIRM demonstrates our commitment to this partnership by funding translational research led by Anthony J. Aldave, MD at UCLA Health for Congenital Hereditary Endothelial Dystrophy (CHED), a corneal condition affecting children. CIRM recently awarded a $4 million grant to?Elpida Therapeutics?to advance a gene therapy for Charcot-Marie-Tooth disease type 4J (CMT4J), a rare, debilitating, progressive hereditary motor and sensory neurological disease.?
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?? On this Rare Disease Day, we honor the resilience and strength of individuals and families living with rare diseases worldwide. At Elpida Therapeutics, we're committed to driving innovation and advancing research to bring hope and support to those in need. Together, let's raise awareness, inspire action, and make a difference. #RareDiseaseDay #ShowYourRare #ElpidaTherapeutics #RareDiseaseAwareness
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We are so very humbled and grateful to CIRM for awarding us this incredible $4 million dollar grant. This critical funding will be used towards IND enabling studies to expedite a gene therapy to patients with CMT4J disease. https://lnkd.in/gXthBnCq We would like to thank our incredible partners for supporting us and helping us work towards a treatment for this orphan disease population. Together we will give children living with this terrible disease a better life! Our Partners California Institute for Regenerative Medicine (CIRM) National Center for Advancing Translational Sciences (NCATS) Foundation for the National Institutes of Health CureCMT4J/Talia Duff Foundation Viralgen Vector Core Charles River Laboratories QuatreLabs Clario KO Law Firm Ciitizen DSST UT Southwestern Medical Center Children's Health Stanford University UIowa Neuroscience Institute National Institute of Neurological Disorders and Stroke (NINDS) Columbus Children's Foundation Fundación Columbus #genetherapy , #ClincialTrials, #CTM4J, #Hope
Elpida Therapeutics Awarded $4M in Grant Funding from The California Institute for Regenerative Medicine (CIRM)
elpidatx.com
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???Exciting News! We are looking forward to?exploring our new?partnerships?with Ciitizen?for both CMT4J and SPG50 programs. Their commitment to enhancing patient-directed healthcare, particularly for those living with rare and complex diseases, is truly inspiring. It's time for novel solutions, and together, we can make significant strides in treating rare conditions.
Today marks an important and exciting milestone for Ciitizen as we announce our divestiture from Invitae and the formation of a new independent company to further our mission. I have never felt prouder to be part of a journey inspired by the thousands of patients and families we stand alongside, as we strive to accelerate drug development and illuminate the best treatment and support options for patients facing rare and complex conditions. We’re grateful to Invitae for their partnership over the years, as well as to our investors, dedicated partners, and above all the patients we serve. But most of all, I am deeply grateful for the remarkable team that has propelled Ciitizen to where we stand today, and the inspired individuals who will continue to carry our mission forward. While we've made significant progress, we are only just getting started!
Ciitizen Forges New Path As Independent Company
Farid Vij，发布于领英
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Big News from Elpida Therapeutics! ? We're excited to announce the launch of a Natural History Study in Charcot-Marie-Tooth Disease Type 4J (CMT4J) by Elpida Therapeutics. This important study, enrolling 20 participants, aims to deepen our understanding of CMT4J and pave the way for future gene therapy trials. ? This initiative underscores our commitment to pioneering healthcare innovation and offering new hope to those affected by this rare condition! https://lnkd.in/geGhuc_s Foundation for the National Institutes of Health,?The National Institutes of Health,?UT Southwestern Medical Center,?Iowa State University,?Stanford University, CureCMT4J/Talia Duff Foundation
Elpida Therapeutics Llaunches Natural History Study In Charcot Marie Tooth Disease Type 4J ?
elpidatx.com