Galactosylceramidase

Passage Bio Doses First Patient in Global Clinical Trial for Infantile Krabbe Disease, A Rare Fatal Pediatric Condition

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Thursday, March 10, 2022
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It is gratifying to dose the first patient in our GALax-C trial, said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio.

Key Points: 
  • It is gratifying to dose the first patient in our GALax-C trial, said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio.
  • Krabbe disease is a rare pediatric lysosomal storage disorder caused by mutations in the GALC gene, which encodes galactosylceramidase, an enzyme that breaks down galactosylceramide and psychosine.
  • We also are grateful to the children, families, and clinical trial investigators who have chosen to participate in our studies.
  • The U.S. Food and Drug Administration (FDA) has granted PBKR03 Fast Track, Orphan Drug, and Rare Pediatric Disease designations.

Gain Therapeutics Progresses Krabbe Disease Program and Provides Scientific Update

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Tuesday, December 7, 2021
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BETHESDA, Md., Dec. 07, 2021 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (Gain, or the Company), a biotechnology company focused on identifying and optimizing allosteric binding sites never before targeted in neurodegenerative diseases and lysosomal storage disorders, today announced a scientific update on the companys Krabbe disease program.

Key Points: 
  • BETHESDA, Md., Dec. 07, 2021 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (Gain, or the Company), a biotechnology company focused on identifying and optimizing allosteric binding sites never before targeted in neurodegenerative diseases and lysosomal storage disorders, today announced a scientific update on the companys Krabbe disease program.
  • Gain is developing allosteric regulators to stabilize the galactosylceramidase (GALC) enzyme and reduce psychosine disease causing toxic substrate, potentially providing the first treatment option to patients with this devastating disease.
  • These data provide additional validation of our approach in treating lysosomal storage diseases, said Eric Richman, Chief Executive Officer of Gain.
  • Krabbe disease, also known as globoid cell leukodystrophy, is a genetic disease that affects the central and peripheral nervous systems.

Forge Biologics Announces Regulatory Updates from FDA and EMA, Accelerating Manufacturing and Clinical Trial Momentum

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Monday, September 20, 2021
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FBX-101 previously received Orphan Drug and Rare Pediatric Disease Designations from the FDA earlier this year.

Key Points: 
  • FBX-101 previously received Orphan Drug and Rare Pediatric Disease Designations from the FDA earlier this year.
  • Forge presented information to the FDA on its proprietary HEK293 suspension cell line, Ignition Cells, and adenovirus helper plasmid, pEMBR, and received FDA alignment that these technologies are suitable for cGMP manufacturing of clinical drug products.
  • The Hearth is a custom-designed cGMP facility dedicated to AAV vector manufacturing and will host end-to-end manufacturing services to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing.
  • By taking a patients-first approach, Forge aims to accelerate the timelines of these transformative medicines for those who need them the most.

Gain Therapeutics, Inc. Reports Second Quarter 2021 Financial Results and Business Update

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Wednesday, August 11, 2021
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BETHESDA, Md., Aug. 11, 2021 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a biotechnology company focused on identifying and optimizing allosteric binding sites that have never been targeted in neurodegenerative diseases and lysosomal storage disorders, today announced its financial results as of and for the second quarter June 30, 2021 and highlighted recent corporate accomplishments.

Key Points: 
  • GAIN continues to expand its intellectual property portfolio and in June announced the publication of two PCT patents.
  • Signed Multi-Target Drug Discovery Collaboration with Zentalis Pharmaceuticals in April to discover new product candidates for the treatment of cancer.
  • As of June 30, 2021, the Companys cash position was $43.16 million, compared to $7.49 million as of December 31, 2020.
  • Cash and cash equivalents were $43.16 million as of June 30, 2021 compared to $7.49 million at December 31, 2020.

FDA Clears IND Application for Passage Bio’s Gene Therapy Candidate PBKR03 for Treatment of Patients with Early Infantile Krabbe Disease, A Rare Pediatric Disorder with No Approved Disease-Modifying Treatment Options

Retrieved on: 
Monday, February 8, 2021
Rare diseases, Lipid storage disorders, Krabbe disease, Leukodystrophies, Organ systems, Galactosylceramidase, Health, Krabbe, Medicine

Underscoring the urgent medical need in the patient population, the FDA has previously granted Passage Bio both Orphan Drug and Rare Pediatric Disease designations for PBKR03 for treatment in Krabbe disease.

Key Points: 
  • Underscoring the urgent medical need in the patient population, the FDA has previously granted Passage Bio both Orphan Drug and Rare Pediatric Disease designations for PBKR03 for treatment in Krabbe disease.
  • PBKR03 utilizes a next-generation proprietary AAV capsid to deliver, through intra-cisterna magna (ICM) administration, a functional GALC gene to Krabbe patients with mutations in the gene that codes for galactosylceramidase (GAL-C).
  • PBKR03 has the potential to treat both the central nervous system and peripheral nerve manifestations observed in Krabbe disease patients.
  • The trial is designed as a dose escalation study of a single ICM dose of PBKR03 in pediatric subjects with early infantile Krabbe disease.

Passage Bio Announces Presentation of Data from Animal Models of Krabbe Disease at the American Society of Gene & Cell Therapy (ASGCT) 23rd Annual Meeting

Retrieved on: 
Tuesday, May 12, 2020
Branches of biology, Neurological disorders, Rare diseases, Enzymes, Lipid storage disorders, Krabbe disease, Leukodystrophies, Galactosylceramidase, Psychosine, Krabbe, Lysosomal storage disease, Gene therapy

This data was presented today in an virtual oral presentation at the American Society of Gene and Cell Therapy (ASGCT) 23rd Annual Meeting by Juliette Hordeaux, D.V.M., Ph.D., senior director of translational research at the University of Pennsylvanias Gene Therapy Program.

Key Points: 
  • This data was presented today in an virtual oral presentation at the American Society of Gene and Cell Therapy (ASGCT) 23rd Annual Meeting by Juliette Hordeaux, D.V.M., Ph.D., senior director of translational research at the University of Pennsylvanias Gene Therapy Program.
  • We are extremely excited about this data for our Krabbe program.
  • Krabbe disease is a rare and often life-threatening lysosomal storage disease caused by mutations in the GALC gene, which encodes galactosylceramidase, an enzyme that breaks down galactosylceramide and psychosine.
  • PBKR03 thus has the potential to treat both the central nervous system and peripheral nerve manifestations observed in Krabbe disease patients.