CLN3

M6P Therapeutics to Present at the 17th Annual International Congress on Neuronal Ceroid Lipofuscinosis

Retrieved on: 
Friday, October 8, 2021

Neuronal ceroid lipofuscinosis type 2 (CLN2) is a neurodegenerative disorder caused by autosomal recessive mutations in the TPP1 gene, leading to the deficiency of TPP1, a lysosomal enzyme.

Key Points: 
  • Neuronal ceroid lipofuscinosis type 2 (CLN2) is a neurodegenerative disorder caused by autosomal recessive mutations in the TPP1 gene, leading to the deficiency of TPP1, a lysosomal enzyme.
  • Batten disease originally referred specifically to the juvenile and most common form of neuronal ceroid lipofuscinosis (NCL), now known as CLN3 .
  • Most forms are inherited in an autosomal recessive manner; however, autosomal dominant inheritance has been reported in one adult-onset form (neuronal ceroid lipofuscinosis 4B).
  • M6P Therapeutics mission is to translate advanced science into best-in-class therapies that address unmet needs within the LSD community.

Amicus Therapeutics and ARYA Sciences Acquisition Corp IV Announce Formation of a Next-Generation Genetic Medicine Company: “Caritas Therapeutics”

Retrieved on: 
Wednesday, September 29, 2021

ET

Key Points: 
  • ET
    PHILADELPHIA & NEW YORK, Sept. 29, 2021 (GLOBE NEWSWIRE) -- Amicus Therapeutics, Inc. (Nasdaq: FOLD) (Amicus) today announced its intent to launch a next-generation genetic medicine company, Caritas Therapeutics, Inc., (Caritas) through a definitive business combination agreement pursuant to which the Amicus gene therapy business will be acquired by ARYA Sciences Acquisition Corp IV (Nasdaq: ARYD) (ARYA IV), a special purpose acquisition company or SPAC, sponsored by Perceptive Advisors.
  • In a single stroke with the formation and funding of Caritas we will create what will be one of the worlds preeminent next-generation genetic medicines companies.
  • Amicus focus will be to grow its leadership position across Amicus two lead indications of Fabry disease and Pompe disease.
  • A webcast of the Caritas Therapeutics presentation materials will also be available on Deal Roadshow:
    Recorded Deal Roadshow Investor Login Details:

Exicure, Inc. Reports Second Quarter 2021 Financial Results and Corporate Progress

Retrieved on: 
Thursday, August 12, 2021

Exicure, Inc. (NASDAQ:XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA) technology, today reported financial results for the quarter ended June 30, 2021 and provided an update on corporate progress.

Key Points: 
  • Exicure, Inc. (NASDAQ:XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA) technology, today reported financial results for the quarter ended June 30, 2021 and provided an update on corporate progress.
  • Interim results from the Phase 2 portion of the clinical trial were reported on August 5, 2021 on 26 patients, 17 of whom were evaluable.
  • Research and Development (R&D) Expenses: R&D expenses were $10.8 million for the quarter ended June 30, 2021, compared to $7.0 million for the quarter ended June 30, 2020.
  • General and Administrative Expenses: General and administrative expenses were $3.1 million for the quarter ended June 30, 2021, compared to $2.2 million for the quarter ended June 30, 2020.

Exicure Announces Upcoming Neuroscience Pipeline Update at Virtual R&D Day

Retrieved on: 
Thursday, July 8, 2021

The event will showcase Exicures neuroscience pipeline, including its lead program for Friedreichs Ataxia (FA), XCUR-FXN, which is designed to address the underlying molecular cause of FA.

Key Points: 
  • The event will showcase Exicures neuroscience pipeline, including its lead program for Friedreichs Ataxia (FA), XCUR-FXN, which is designed to address the underlying molecular cause of FA.
  • Exicure will present new and previously unreleased preclinical data and discuss progress with XCUR-FXN, which is on track for IND filing in late Q4 2021.
  • Additionally, Exicure will discuss its perspective on XCUR-FXNs competitive differentiation in FA and the programs path to clinical validation.
  • Also, Exicure will provide an update on its expanding pipeline across several rare neurodegenerative diseases of high unmet need and highlight progress with two preclinical programs targeting SCN9A (Nav1.7) for neuropathic pain and CLN3 for Batten Disease.

Beyond Batten Disease Foundation Announces Agreement with Actelion Pharmaceuticals Ltd to Provide Drug Product for BBDF-101

Retrieved on: 
Tuesday, March 2, 2021

Trehalose is a naturally occurring disaccharide which is Generally Regarded as Safe (GRAS) by the US Federal Drug Administration (FDA).

Key Points: 
  • Trehalose is a naturally occurring disaccharide which is Generally Regarded as Safe (GRAS) by the US Federal Drug Administration (FDA).
  • These data prompted BBDF to provide the financing to support the development of BBDF-101 as a potential treatment of CLN3 disease.
  • "We are incredibly grateful to Janssen for its support for the development of BBDF-101 for Batten Disease.
  • Beyond Batten Disease Foundation (BBDF) is the world's largest non-profit organization dedicated to funding research for a treatment and cure for juvenile (CLN3) Batten disease.

Beyond Batten Disease Foundation Announces Agreement with Actelion Pharmaceuticals Ltd to Provide Drug Product for BBDF-101

Retrieved on: 
Tuesday, March 2, 2021

Trehalose is a naturally occurring disaccharide which is Generally Regarded as Safe (GRAS) by the US Federal Drug Administration (FDA).

Key Points: 
  • Trehalose is a naturally occurring disaccharide which is Generally Regarded as Safe (GRAS) by the US Federal Drug Administration (FDA).
  • These data prompted BBDF to provide the financing to support the development of BBDF-101 as a potential treatment of CLN3 disease.
  • "We are incredibly grateful to Janssen for its support for the development of BBDF-101 for Batten Disease.
  • Beyond Batten Disease Foundation (BBDF) is the world's largest non-profit organization dedicated to funding research for a treatment and cure for juvenile (CLN3) Batten disease.

Amicus Therapeutics Announces Positive Initial Clinical Data for CLN3 Batten Disease Gene Therapy at the 17th Annual WORLDSymposium™ 2021

Retrieved on: 
Monday, February 8, 2021

PHILADELPHIA, Feb. 08, 2021 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced positive initial results from its first in-human study of its CLN3 Batten disease gene therapy program, AT-GTX-502.

Key Points: 
  • PHILADELPHIA, Feb. 08, 2021 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced positive initial results from its first in-human study of its CLN3 Batten disease gene therapy program, AT-GTX-502.
  • The results are featured in a virtual poster presentation at the 17th Annual WORLDSymposium 2021, being held February 8-12, 2021.
  • Initial safety data are available for the first four children up to 15 months post-administration of the AAV-CLN3 gene therapy.
  • Jeff Castelli, Ph.D., Chief Development Officer ofAmicus Therapeutics, stated, We are pleased to share this first set of clinical data for our intrathecal AAV gene therapy for CLN3 Batten disease and the second clinical program in our Batten portfolio.

Polaryx Therapeutics Receives FDA Fast Track Designation to PLX-200 for the Treatment of Patients With Juvenile Neuronal Ceroid Lipofuscinosis

Retrieved on: 
Thursday, August 20, 2020

PARAMUS, N.J., Aug. 20, 2020 /PRNewswire/ --Polaryx Therapeutics Inc., a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to PLX-200 for the treatment of Juvenile Neuronal Ceroid Lipofuscinosis (JNCL/CLN3) patients.

Key Points: 
  • PARAMUS, N.J., Aug. 20, 2020 /PRNewswire/ --Polaryx Therapeutics Inc., a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to PLX-200 for the treatment of Juvenile Neuronal Ceroid Lipofuscinosis (JNCL/CLN3) patients.
  • It is the most prevalent among the NCL (neuronal ceroid lipofuscinosis) diseases and occurs in 3 out of 100,000 births.
  • "We are very excited about the FDA Fast Track Designation to PLX-200 for CLN3 treatment.
  • This Fast Track Designation will expedite PLX-200 development process for the treatment of CLN3 patientswith morefrequent accesses to the FDA.

Polaryx Therapeutics Receives IND Approval From the FDA to Study PLX-200 Treatment for Patients With Juvenile Neuronal Ceroid Lipofuscinosis

Retrieved on: 
Wednesday, April 8, 2020

PARAMUS, N.J., April 8, 2020 /PRNewswire/ -- Polaryx Therapeutics, a biotech company developing patient-friendly, small molecule therapeutics for lysosomal storage disorders, announced today that the Company has received an Investigational New Drug Application (IND) approval from the U.S. Food and Drug Administration (FDA) to study PLX-200 treatment on Juvenile Neuronal Ceroid Lipofuscinosis (JNCL or CLN3) patients.

Key Points: 
  • PARAMUS, N.J., April 8, 2020 /PRNewswire/ -- Polaryx Therapeutics, a biotech company developing patient-friendly, small molecule therapeutics for lysosomal storage disorders, announced today that the Company has received an Investigational New Drug Application (IND) approval from the U.S. Food and Drug Administration (FDA) to study PLX-200 treatment on Juvenile Neuronal Ceroid Lipofuscinosis (JNCL or CLN3) patients.
  • "We are very excited about our CLN3 IND approval from the FDA, as we can go ahead with CLN3 clinical studies with PLX-200.
  • Polaryx Therapeutics, Inc is dedicated to developing drug candidates for lysosomal storage disorders, for which there is currently no safe and patient-friendly treatment option available.
  • Polaryx is repurposing existing safe oral medications and/or developing new drugs, so that the treatment is patient-friendly for a prolonged use.

Rare disease in children: the key role of a protein revealed

Retrieved on: 
Thursday, February 27, 2020

Children affected by Batten disease are born with no symptoms and develop normally, learning to walk, talk, and interact with others.Between 5 and 8years of age, however, they start to regress.

Key Points: 
  • Children affected by Batten disease are born with no symptoms and develop normally, learning to walk, talk, and interact with others.Between 5 and 8years of age, however, they start to regress.
  • Professor Lefranois is working with a team of European researchers to re-establish normal CLN3 function with a promising drug.
  • Worldwide, it is estimated that one person in 100,000 has Batten's disease in all its forms.
  • The Institut National de la Recherche Scientifique (INRS) is the only institution in Qubec dedicated exclusively to graduate level university research and training.