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Home » amyotrophic lateral sclerosis

Articles Tagged with ''amyotrophic lateral sclerosis''

Neurology/psychiatric

Voyager to evaluate alternate payloads for ALS gene therapy program

Feb. 12, 2025
Voyager Therapeutics Inc. has announced its decision to assess alternate payloads related to its gene therapy program for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). Emerging preclinical data indicate the siRNA payload component of VY-9323 does not meet its standards due to what appears to be an off-target effect resulting in a narrowed therapeutic window.
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Degradation of motor neurons
Neurology/psychiatric

Lifearc and Neuropeutics collaborate to advance small molecule for ALS

Jan. 28, 2025
Lifearc has announced a collaboration with Neuropeutics Inc. to develop a new small molecule for the treatment of motor neuron disease (MND), or amyotrophic lateral sclerosis (ALS). Neuropeutics and Lifearc intend to develop a lead candidate therapeutic to prevent and reverse TDP-43 protein aggregation in MND/ALS.
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Illustration of double helix
Newco news

Taking on gene delivery, Coave raises $33M series A

Jan. 9, 2025
By Nuala Moran
Coave Therapeutics SA has completed the transition to becoming a genetic medicines specialist, after divesting its single ophthalmology program and raising $33 million in a series A round.
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Celosia team
Neurology/psychiatric

Australia’s Celosia heads toward clinic with gene therapy for ALS

Jan. 8, 2025
By Tamra Sami
After raising AU$16.75 million (US$10.4 million) in a series A round, Celosia Therapeutics Pty Ltd. is heading toward the clinic with its novel gene therapy that targets TDP-43, a protein directly linked to amyotrophic lateral sclerosis (ALS) pathology.
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Celosia team
Newco news

Australia’s Celosia heads toward clinic with gene therapy for ALS

Jan. 7, 2025
By Tamra Sami
After raising AU$16.75 million (US$10.4 million) in a series A round, Celosia Therapeutics Pty Ltd. is heading toward the clinic with its novel gene therapy that targets TDP-43, a protein directly linked to amyotrophic lateral sclerosis (ALS) pathology.
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Celosia team
Newco news

Australia’s Celosia heads toward clinic with gene therapy for ALS

Jan. 3, 2025
By Tamra Sami
After raising AU$16.75 million (US$10.4 million) in a series A round, Celosia Therapeutics Pty Ltd. is heading toward the clinic with its novel gene therapy that targets TDP-43, a protein directly linked to amyotrophic lateral sclerosis (ALS) pathology.
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Central nervous system
Neurology/psychiatric

Bristol Myers Squibb selects novel ALS target under Insitro collaboration

Dec. 19, 2024
Bristol Myers Squibb Co. has selected the first novel target for amyotrophic lateral sclerosis (ALS) that was identified and validated by Insitro Inc. under the companies’ 2020 collaboration agreement to discover new therapies for ALS. The selection triggers a milestone payment to Insitro.
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AI-generated illustration of spinal cord

Dewpoint and Mitsubishi ink $480M ALS research collaboration

Dec. 10, 2024
By Tamra Sami
Dewpoint Therapeutics Inc. and Mitsubishi Tanabe Pharma Corp. have entered a research collaboration worth up to $480 million to advance Dewpoint’s novel TDP-43 small-molecule condensate modulator for amyotrophic lateral sclerosis. Under terms of the deal, Boston-based Dewpoint will receive an undisclosed up-front payment and is eligible to receive R&D-based milestone payments up to $480 million. Upon reaching those milestones, Osaka, Japan-based MTPC will have an exclusive option to license the program and assume responsibility for global clinical development and commercialization. Dewpoint will also receive tiered royalties on net sales.
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AI-generated illustration of spinal cord
Neurology/psychiatric

Dewpoint and Mitsubishi ink $480M ALS research collaboration

Dec. 10, 2024
By Tamra Sami
Dewpoint Therapeutics Inc. and Mitsubishi Tanabe Pharma Corp. have entered a research collaboration worth up to $480 million to advance Dewpoint’s novel TDP-43 small-molecule condensate modulator for amyotrophic lateral sclerosis. Under terms of the deal, Boston-based Dewpoint will receive an undisclosed up-front payment and is eligible to receive R&D-based milestone payments up to $480 million. Upon reaching those milestones, Osaka, Japan-based MTPC will have an exclusive option to license the program and assume responsibility for global clinical development and commercialization. Dewpoint will also receive tiered royalties on net sales.
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Neurology/psychiatric

Equity investment in Eikonizo supports HDAC6 inhibitor work

Dec. 10, 2024
Eikonizo Therapeutics Inc. has secured an equity investment from Novo Nordisk A/S.
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