An opportunity to make a difference

Our goal is to transform the lives of patients with rare genetic disease. By intervening early, we believe we will be able to restore the function of aberrant genes before the patients experience irreversible declines in function.

Our technology platform is modular in nature; our initial product candidates will leverage our existing knowledge to address diseases by targeting the liver, including a category of diseases known as inborn errors of metabolism. We expect that our future product candidates will also draw from programs that address genetic diseases by targeting other tissues including the central nervous system (CNS) and muscle.

Disease focus

Our focus on metabolic diseases has led us to intensive study of organic acidemias, which are caused by an enzymatic defect in the metabolism of amino acids, leading to a dangerously high level of organic acid in the blood and body tissues. We are pursuing methylmalonic acidemia (MMA) as an initial indication. Learn more about our therapeutic focus:

Pipeline

LogicBio Publications

Lisowski L et al. Selection and evaluation of clinically relevant AAV variants in a xenograft liver model. Nature. 2014 Feb 20;506(7488):382-6. Epub 2013 Dec 25.

Barzel A, et al. Promoterless gene targeting without nucleases ameliorates haemophilia B in mice. Nature. 2015 Jan 15;517(7534):360-4. Epub 2014 Oct 29.

Nygaard S, et al. A universal system to select gene-modified hepatocytes in vivo. Sci Transl Med. 2016 Jun 8;8(342):342ra79.

Porro F, et al. Promoterless gene targeting without nucleases rescues lethality of a Crigler-Najjar syndrome mouse model. EMBO Mol Med. 2017 Oct;9(10):1346-1355.

Borel F, et al. Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency. Mol Ther. 2017 Nov 1;25(11):2477-2489. Epub 2017 Sep 25.

Supporting Publications

Wang P, et al. Induction of hepatocellular carcinoma by in vivo gene targeting. Proceedings of the National Academy of Sciences of the United States of America. 2012 Jul 10;109(28):11264-9. Epub 2012 Jun 25.

Chandler R, et al. Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy. Journal of Clinical Investigation. 2015 Feb;125(2):870-80. Epub 2015 Jan 20.

Carroll D. Staying on target with CRISPR-Cas. Nature Biotechnology. 2013 Sep;31(9):807-9.

Kosicki M, et al. Repair of double-strand breaks induced by CRISPR-Cas9 leads to large deletions and complex rearrangements. Nature Biotechnology. 2018 Sep;36(8):765-771. Epub 2018 Jul 16.

Melo S, et al. Somatic correction of junctional epidermolysis bullosa by a highly recombinogenic AAV variant. Molecular Therapy. 2014 Apr;22(4):725-33. Epub 2014 Jan 6.

Sebastiano V, et al. Human COL7A1-corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosa. Science Translational Medicine. 2014 Nov 26;6(264):264ra163.

Liu Z, et al. Systematic comparison of 2A peptides for cloning multi-genes in a polycistronic vector. Scientific Reports. 2017 May 19;7(1):2193.

Qasim W, et al. Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells. Science Translational Medicine. 2017 Jan 25;9(374).

Wang L, et al. Hepatic gene transfer in neonatal mice by adeno-associated virus serotype 8 vector. Human Gene Therapy. 2012 May;23(5):533-9. Epub 2018 Feb 8.

Wang L, et al. AAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta). Molecular Therapy. 2011 Nov;19(11):2012-20. Epub 2011 Aug 2.

Simhadri V, et al. Prevalence of Pre-existing Antibodies to CRISPR-Associated Nuclease Cas9 in the USA Population. Molecular therapy. Methods & clinical development. 2018 Jun 15;10:105-112. eCollection 2018 Sep 21.