The initial development programs will focus on genetic Parkinson’s disease (PD) and Friedreich’s ataxia (FRDA). These diseases have a high unmet medical need and known genetic causes. The target genes, 33kb and 135kb, respectively, far exceed the 5kb payload capacity of adeno-associated virus (AAV) vectors.
Wednesday, December 14, 2022
Kaleibe launched by Replay to target genetic brain disorders with gene therapy
14-12-2022. Replay, a genome writing company reprogramming biology by writing and delivering big DNA, has announced the launch of Kaleibe, a herpes simplex virus (HSV) gene therapy company targeting genetic brain disorders.
It is the third product company to launch since Replay’s formation in July and will leverage its high payload capacity HSV delivery vector, synHSVTM, to target genetic brain disorders.
G-rich motifs within phosphorothioate-based antisense oligonucleotides (ASOs) drive activation of FXN expression through indirect effects
Feng Wang, Ezequiel Calvo-Roitberg, Julia M Rembetsy-Brown, Minggang Fang, Jacquelyn Sousa, Zachary J Kartje, Pranathi Meda Krishnamurthy, Jonathan Lee, Michael R Green, Athma A Pai, Jonathan K Watts; Nucleic Acids Research, 2022;, gkac1108, doi:10.1093/nar/gkac1108
The phosphorodiamidate morpholino oligomer analogs of our ASOs did not activate FXN, pointing to a PS-backbone-mediated effect. Our study demonstrates the importance of multiple, detailed control experiments and target validation in oligonucleotide studies employing novel mechanisms such as gene activation.
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