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Neurogene Announces FDA Orphan Drug Designation for CLN7 Batten Disease Gene Therapy

https://www.businesswire.com/news/home/20200811005348/en/

https://www.neurogene.com/press-releases/neurogene-announces-fda-orphan-drug-designation-for-cln7-batten-disease-gene-therapy/

Neurogene Announces FDA Orphan Drug Designation for CLN7 Batten Disease Gene Therapy
FDA grants Orphan Drug Designation to Neurogene’s adeno-associated virus vector with engineered transgene encoding the human CLN7 gene

August 11, 2020 09:00 AM Eastern Daylight Time
NEW YORK–(BUSINESS WIRE)–Neurogene Inc., a company founded with a mission to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to adeno-associated virus vector with engineered transgene encoding the human CLN7 gene for patients with CLN7, a form of Batten disease. Batten disease, a common name for a rare class of diseases called neuronal ceroid lipofuscinoses (NCLs), affects an estimated 2-4 out of every 100,000 children in the United States. In July, the company announced it had received Orphan Drug Designation from the FDA for its gene therapy for the treatment of CLN5 Batten disease.

Exon skipping combats Batten disease

https://www.nature.com/articles/d41573-020-00136-7

https://doi.org/10.1038/d41573-020-00136-7

https://pubmed.ncbi.nlm.nih.gov/32753718/

TITLE:
Exon skipping combats Batten disease

ALTERNATIVE TITLE:
None

DATE:
Wed, 05 Aug 2020 06:00:00 -0400

AUTHORS:
Sarah Crunkhorn

SOURCE:
Nature reviews. Drug discovery

DESCRIPTION:
No abstract

CONTENT:
Nat Rev Drug Discov. 2020 Aug 4. doi: 10.1038/d41573-020-00136-7. Online ahead of print.

NO ABSTRACT

PMID:32753718 | DOI:10.1038/d41573-020-00136-7

PUBMED ID:
pubmed:32753718

OTHER ID:
pmid:32753718,doi:10.1038/d41573-020-00136-7

PUBLICATION DATE:
Wed, 05 Aug 2020 06:00:00 -0400
2020-08-06

RETRIEVAL DATE :
08/07/20 03:21PM

LINK – PUBMED:
https://pubmed.ncbi.nlm.nih.gov/32753718/?utm_source=MS-Office&utm_medium=rss&utm_campaign=pubmed-2&utm_content=1nWYA0Nnwr_wLHWsIDX2btUvlzF91Kc2m_rAz6Uc2Ke1bh4s-l&fc=20200622115739&ff=20200805063026&v=2.11.4

LINK – DOI:
https://doi.org/10.1038/d41573-020-00136-7

LINK – FULL TEXT:
Pending

NOTES:
None

Rosalind Franklin University Researcher awarded NIH grant for CLN3 Batten disease study

https://www.eurekalert.org/pub_releases/2020-07/rfuo-rfu073120.php

NEWS RELEASE 31-JUL-2020
Rosalind Franklin University Researcher awarded NIH grant for CLN3 Batten disease study
Michelle Hastings, Ph.D., director of the Center for Genetic Diseases, is leading an NIH-funded team in the study of a new therapeutic approach for CLN3 Batten disease in children; study published July 27, Nature Medicine

ROSALIND FRANKLIN UNIVERSITY OF MEDICINE AND SCIENCE

Research reveals key role of a protein in rare Batten disease

https://www.news-medical.net/news/20200228/Research-reveals-key-role-of-a-protein-in-rare-Batten-disease.aspx

Research reveals key role of a protein in rare Batten disease
Download PDF Copy
Reviewed by James Ives, M.Psych. (Editor)Feb 28 2020
Professor Stéphane Lefrançois, a researcher at the Institut National de la Recherche Scientifique (INRS), is working on Batten disease, a neurodegenerative genetic disease that primarily affects children. His research focuses on the most common form of the disease – Batten CLN 3 – which is caused by mutations in the protein of the same name and for which there is still no cure.

CLN3

Theranexus and Beyond Batten Disease Foundation (BBDF) announce signing of exclusive license for Batten drug candidate BBDF-101 [PDF]

https://www.theranexus.com/images/pdf/Theranexus_PR_signature_Accord_BBDF_VUKDEF.pdf

Theranexus and Beyond Batten Disease Foundation (BBDF) announce the signing of a worldwide exclusive license for Batten disease drug candidate BBDF-101

  • The agreement covers the development and marketing of the drug following a single clinical trial due to begin in 2020.
  • If successful, this trial could lead directly to the drug’s approval.

Lyon, 12 December 2019 – Theranexus, a biopharmaceutical company innovating in the treatment of neurological diseases and pioneer in the development of drug candidates modulating the interaction between neurons and glial cells, is pleased to announce the signing of an agreement with Beyond Batten Disease Foundation, granting it a worldwide exclusive license to develop and commercialize the drug candidate BBDF-101 for juvenile Batten disease.

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