CLN1


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AcronymDefinition
CLN1Ceroid Lipofuscinosis Neuronal 1
References in periodicals archive ?
June 18, 2019: Received FDA Fast Track Designation for ABO-202 AAV9 gene therapy in CLN1 disease
New data from IND-enabling studies of our program in CLN1 disease, expected to enter the clinic later this year, will also be presented, said Joo Siffert, M.D., Chief Executive Officer.
This designation builds on our clinical portfolio of AAV gene therapies that have received FDA and EMA orphan drug designations, which is an important validation of the scientific and clinical translation of these products for the severely underserved CLN1 patient population, stated Timothy J.
cerevisiae enhances expression of ribosomal protein genes, glycolytic genes, and all three G1 cyclin genes (CLN1, CLN2, and CLN3).
DBS were obtained from 6 patients with CLN1, 5 patients with CLN2, and 2 patients with the juvenile form CLN3, as well as from 70 control individuals.
The absence of this enzyme in patients with CLN1 disease results in malfunctioning cells, including brain cells, neuroinflammation, and neurodegeneration.
Under the terms of the agreement, REGENXBIO has granted Abeona an exclusive worldwide license (subject to certain non-exclusive rights previously granted for MPS IIIA), with rights to sublicense, to REGENXBIOs NAV AAV9 vector for the development and commercialization of gene therapies for the treatment of MPS IIIA, MPS IIIB, CLN1 Disease and CLN3 Disease.
Under the terms of the agreement, REGENXBIO has granted Abeona an exclusive worldwide license with rights to sublicense, to REGENXBIO's NAV AAV9 vector for the development and commercialization of gene therapies for the treatment of MPS IIIA, MPS IIIB, CLN1 Disease and CLN3 Disease.
Key findings included: CLN1 mice recapitulate the major features of the human disease manifestations; The data demonstrate that a single intrathecal (IT) injection of self-complementary adeno-associated virus 9 (scAAV9) encoding the human CLN1 gene to CLN1 mice at 1 week and 1 month (pre-symptomatic) significantly increased their survival, improved behavior and reduced motor deficits.
The neuronal ceroid lipofuscinoses (NCLs) are a group of inherited neurodegenerative diseases that include infantile NCL (INCL; OMIM 256730, where the defective gene is CLN1), classical late infantile NCL (LINCL; OMIM 204500, where the defective gene is CLN2), two variant late infantile NCLs (OMIM 256731, where the defective gene is CLN5; and OMIM 601780, where the defective gene is CLN6), juvenile NCL (JNCL; OMIM 204200; defective gene, CLN3), a juvenile onset epilepsy with progressive mental retardation (EPMR; OMIM 600143; defective gene, CLN8), and adult NCL (Kufs disease; OMIM 204300; defective gene, CLN4) [reviewed in Ref.
ABO-202 is an adeno-associated virus developed to introduce a functional copy of the CLN1 gene into cells in order to restore the enzyme activity that is needed to break down certain lipopigment proteins that are deficient in patients with CLN1 disease.
We are excited to celebrate Abeonas first R&D day this fall and update our stakeholders on the significant progress made in our clinical stage gene therapy programs for ABO-102 and EB-101, as well as provide a review of the progress made on the IND-enabling studies for our juvenile (CLN3) and infantile (CLN1) Batten programs, stated Timothy J.