Plasma multi-omics analysis reveals very long chain ceramides as validated biomarkers of Friedreich’s ataxia

Dezhen Wang, M. Grazia Cotticelli, Blanca E. Himes, David R. Lynch, Clementina Mesaros; medRxiv 2022.09.27.22280432; doi:10.1101/2022.09.27.22280432

 New plasma lipids biomarkers of Friedreich’s Ataxia (FRDA) were validated using a discovery-validation design with two independent cohorts.

Solid Biosciences Announces Acquisition of AavantiBio and Concurrent $75 Million Private Placement

September 30, 2022 07:00 ET | Source: Solid Biosciences Inc CHARLESTOWN, Mass. and CAMBRIDGE, Mass., Sept. 30, 2022 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy (Duchenne), and AavantiBio, Inc., a privately-held gene therapy company focused on transforming the lives of patients with Friedreich’s ataxia and rare cardiomyopathies, today announced that the companies have entered into a definitive merger agreement whereby Solid will acquire AavantiBio, including its pipeline assets and net cash. The combined company will focus on advancing a portfolio of neuromuscular and cardiac programs, led by SGT-003, a differentiated gene transfer candidate, for the treatment of Duchenne. Additional pipeline programs include AVB-202, a gene transfer candidate for the treatment of Friedreich’s ataxia, AVB-401 for BAG3 mediated dilated cardiomyopathy, and additional assets for the treatment of undisclosed cardiac diseases. Following approval by Solid stockholders, the combined company will operate as Solid Biosciences, will trade on Nasdaq under the ticker symbol “SLDB” and Bo Cumbo, the current Chief Executive Officer of AavantiBio, will assume the role of President and CEO of Solid Biosciences. 

FA is a rare inherited neuromuscular disease that causes progressive nervous system damage and movement problems. AVB-202, AavantiBio’s lead AAV gene transfer therapy candidate in preclinical development, utilizes a dual route of administration to more rigorously target disease pathology. Preclinical data from three animal models, including mouse and nonhuman primate, supported preclinical proof of concept. Solid is anticipating an IND submission for AVB-202 in the second half of 2024.

Friedreich Ataxia: An expanded access program for Elamipretide treatment

Children's Mercy Kansas City / Children’s Mercy Research Institute. 

Full Study Name: SPIES-006

This study is for participants who are 1 to 80 years old and involves the treatment use of Elamipretide through the expanded access program for treatment of Friedreich Ataxia. This is a compassionate use study. Compassionate use studies are those where the drug is not available to the market and the sponsor is providing the drug to the patient without cost to them in hopes that it will help decrease the progression of their disease state.