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Digital content
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24 Apr 2025
// PR NEWSWIRE
https://www.prnewswire.com/news-releases/alcyone-therapeutics-announces-strategic-financing-to-advance-an-expanded-pipeline-of-pediatric-focused-precision-therapeutics-for-severe-neurological-diseases-302436946.html
31 Jul 2024
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https://www.prnewswire.com/news-releases/alcyone-therapeutics-announces-continued-enrollment-approval-from-fda-of-the-pierre-pivotal-ide-clinical-study-of-the-thecaflex-drx-system-for-administration-of-nusinersen-302210909.html
08 May 2024
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https://www.prnewswire.com/news-releases/alcyone-therapeutics-announces-appointment-of-dr-norbert-riedel-as-chairman-of-the-board-and-dr-kathrin-meyer-as-chief-scientific-officer-and-head-of-research--development-302139018.html
27 Jun 2023
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https://www.prnewswire.com/news-releases/alcyone-therapeutics-receives-fda-ide-approval-to-initiate-clinical-study-of-the-thecaflex-drxtm-system-for-administration-of-spinraza-nusinersen-301863840.html
02 May 2022
// PRNEWSWIRE
https://www.prnewswire.com/news-releases/alcyone-announces-two-oral-presentations-on-its-gene-therapy-platforms-at-the-25th-american-society-of-gene-and-cell-therapy-asgct-annual-meeting-301537713.html
Details:
The financing aims to advance the clinical developed of company's ealry-stage product CLN-301, which is being evaluated for the treatment of CLN3 Batten disease.
Lead Product(s): CLN-301
Therapeutic Area: Rare Diseases and Disorders Brand Name: CLN-301
Study Phase: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Sponsor: Nationwide Children’s Hospital
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Financing April 24, 2025
Lead Product(s) : CLN-301
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I/ Phase II
Partner/Sponsor/Collaborator : Nationwide Children’s Hospital
Deal Size : Undisclosed
Deal Type : Financing
Alcyone Gains Strategic Funding for Pediatric Neuro Therapies
Details : The financing aims to advance the clinical developed of company's ealry-stage product CLN-301, which is being evaluated for the treatment of CLN3 Batten disease.
Product Name : CLN-301
Product Type : Cell and Gene therapy
Upfront Cash : Undisclosed
April 24, 2025
Details:
SPINRAZA (nusinersen) is an antisense oligonucleotide (ASO) that targets the root cause of SMA by continuously increasing the amount of full-length survival motor neuron (SMN) protein produced in the body.
Lead Product(s): Nusinersen Sodium
Therapeutic Area: Genetic Disease Brand Name: Spinraza
Study Phase: Approved FDFProduct Type: Oligonucleotide
Sponsor: Biogen
Deal Size: Inapplicable Upfront Cash: Inapplicable
Deal Type: Inapplicable June 27, 2023
Lead Product(s) : Nusinersen Sodium
Therapeutic Area : Genetic Disease
Highest Development Status : Approved FDF
Partner/Sponsor/Collaborator : Biogen
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : SPINRAZA (nusinersen) is an antisense oligonucleotide (ASO) that targets the root cause of SMA by continuously increasing the amount of full-length survival motor neuron (SMN) protein produced in the body.
Product Name : Spinraza
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
June 27, 2023
Details:
SPINRAZA (nusinersen) is an antisense oligonucleotide (ASO) that targets the root cause of SMA by continuously increasing the amount of full-length survival motor neuron (SMN) protein produced in the body.
Lead Product(s): Nusinersen Sodium
Therapeutic Area: Genetic Disease Brand Name: Spinraza
Study Phase: Approved FDFProduct Type: Oligonucleotide
Sponsor: Biogen
Deal Size: $51.0 million Upfront Cash: $10.0 million
Deal Type: Collaboration April 01, 2023
Lead Product(s) : Nusinersen Sodium
Therapeutic Area : Genetic Disease
Highest Development Status : Approved FDF
Partner/Sponsor/Collaborator : Biogen
Deal Size : $51.0 million
Deal Type : Collaboration
Details : SPINRAZA (nusinersen) is an antisense oligonucleotide (ASO) that targets the root cause of SMA by continuously increasing the amount of full-length survival motor neuron (SMN) protein produced in the body.
Product Name : Spinraza
Product Type : Oligonucleotide
Upfront Cash : $10.0 million
April 01, 2023
Details:
Updated preclinical safety and efficacy data will be presented for ACTX-401 (AAV9-IGHMBP2 Gene Therapy), a gene replacement therapy currently in a Phase 1/2 study for the treatment of IGHMBP2-related disorders.
Lead Product(s): ACTX-401
Therapeutic Area: Genetic Disease Brand Name: ACTX-401
Study Phase: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Sponsor: Inapplicable
Deal Size: Inapplicable Upfront Cash: Inapplicable
Deal Type: Inapplicable February 05, 2022
Lead Product(s) : ACTX-401
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Partner/Sponsor/Collaborator : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Updated preclinical safety and efficacy data will be presented for ACTX-401 (AAV9-IGHMBP2 Gene Therapy), a gene replacement therapy currently in a Phase 1/2 study for the treatment of IGHMBP2-related disorders.
Product Name : ACTX-401
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
February 05, 2022
Details:
Alcyone’s pipeline currently includes 12 adeno-associated virus (AAV) gene therapy programs for severe CNS disorders. Alcyone’s four gene therapy platform technologies include X-reactivation, conventional transgene replacement, vectorized exon skipping and promotor modulation.
Lead Product(s): ACTX-101
Therapeutic Area: Rare Diseases and Disorders Brand Name: ACTX-101
Study Phase: PreclinicalProduct Type: Cell and Gene therapy
Sponsor: RTW Investments
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Funding September 06, 2021
Lead Product(s) : ACTX-101
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Preclinical
Partner/Sponsor/Collaborator : RTW Investments
Deal Size : Undisclosed
Deal Type : Funding
Alcyone Therapeutics Launches to Advance Next-Generation Gene Therapies for CNS Disorders
Details : Alcyone’s pipeline currently includes 12 adeno-associated virus (AAV) gene therapy programs for severe CNS disorders. Alcyone’s four gene therapy platform technologies include X-reactivation, conventional transgene replacement, vectorized exon skippi...
Product Name : ACTX-101
Product Type : Cell and Gene therapy
Upfront Cash : Undisclosed
September 06, 2021
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