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smahSMARD is proud to make the most exciting announcement yet - that the first ever therapy for IGHMBP2-related disorders has been granted approval for a clinical trial to begin!
In 2018, smashSMARD and smashSMARD.de approached Kathrin Meyer’s Lab at Nationwide Children's Hospital to lead pre-clinical research and development of a clinical grade gene therapy for IGHMBP2-related disorders. Almost 3 years to the day, we are beyond hopeful to share this news.
We are pleased that the U.S. Food and Drug Administration recognizes the significant unmet need for treatments for IGHMBP2-related disorders, including SMARD1 and CMT2S. In October 2021, the FDA cleared the Investigational New Drug (IND) application which allows a clinical trial to begin.
This announcement would not be possible without the collaboration from Gregory Cox at The Jackson Laboratory, Dr. Burghes’ Lab and Dr. Arnold’s Lab at The Ohio State University, and Dr. Corti Lab at Associazione Centro Dino Ferrari. Additionally, thank you to Andelyn Biosciences for timely clinical product development. The FDA played an instrumental role in allowing this program to move forward at lightning speed. Lastly, thank you to the principal investigator and treating physician, Dr. Megan Waldrop at Nationwide Children’s Hospital for your commitment to our community.
In Alcyone Therapeutics Inc., we found a steadfast partner who is funding the clinical trial in addition to significant preclinical development funding including chemistry, manufacturing and control. Their support was not limited to funding but included non-monetary support pertinent to the clinical development.
Thank you most importantly to our tens of thousands of donors who have believed in our cause, the families impacted by this disease. Without you, this treatment and announcement would not exist.
WE ARE ON A MISSION TO FIND A CURE
SMARD stands for Spinal Muscular Atrophy with Respiratory Distress (also known as SMARD1). There is little known about this ultra rare and often fatal disease. Its rarity is of no comfort for the families affected by SMARD, and so we are building a community to promote awareness and research to make a cure a reality.
SmashSMARD is proud to announce its support of a promising research program to be conducted by Dr. Kathrin Meyer at the Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio USA. Dr. Meyer's laboratory at AWRI has a longstanding translational focus on neurodegenerative diseases and an encouraging track record of moving therapeutic programs towards clinical trial.
With SmashSMARD’s funding support, Dr. Meyer is now bringing that focus to SMARD. Dr. Meyer’s lab develops Gene Therapies for neurological and neuromuscular disorders by using an approach that increases full length protein expression.
SmashSMARD looks forward to Dr. Meyer’s work in supporting the translation of SMARD research to a CLINICAL TRIAL IN THE COMING MONTHS.
Gene therapy aims to replace or correct the faulty IGHMBP2 gene and is a well-documented treatment for this disease in animal models. This video shows success of gene therapy in mice at the Univesity of Milan (AAV-null mouse is untreated; AAV-IGHMBP2 has received gene therapy).
We are Funding Research Now for SMARD, CMT2S and IGHMBP2!
Our small SMARD community is committed to spreading the word and doing all we can to get these wonderful kids treatment as soon as possible. For a critically ill individual, everyday is a celebration of life and we plan to celebrate each of these faces for many, many more days and years to come.