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BioWorld - Saturday, January 31, 2026
Home » Topics » Drugs » Gene therapy

Gene therapy
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Colorful illustration of the heart
Cardiovascular

Affinia names development candidate for BAG3 dilated cardiomyopathy

Jan. 10, 2025
Affinia Therapeutics Inc. has nominated AFTX-201 as a development candidate for the treatment of BAG3 dilated cardiomyopathy. The gene therapy, using Affinia’s cardiotropic capsid, is given as a one-time intravenous injection.
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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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Molecules and RNA enclosed by a lipid bilayer
Hematologic

Orna, Vertex next-gen sickle cell, thalassemia deal could reach $4.3B+

Jan. 8, 2025
By Karen Carey
About four years after launch, Orna Therapeutics Inc. signed its second major deal, this time validating the lipid nanoparticle delivery technology it acquired through its Renagade Therapeutics Inc. buyout in May 2024, with Vertex Pharmaceuticals Inc. seeking next-generation approaches for hemoglobinopathies.
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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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Neurology/psychiatric

Solid Biosciences gains IND clearance for Friedreich’s ataxia gene therapy

Jan. 8, 2025
Solid Biosciences Inc. has gained IND clearance from the FDA for SGT-212 for the treatment of Friedreich’s ataxia (FA).
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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.
Read More
Molecules and RNA enclosed by a lipid bilayer

Orna, Vertex next-gen sickle cell, thalassemia deal could reach $4.3B+

Jan. 7, 2025
By Karen Carey
About four years after launch, Orna Therapeutics Inc. signed its second major deal, this time validating the lipid nanoparticle delivery technology it acquired through its Renagade Therapeutics Inc. buyout in May 2024, with Vertex Pharmaceuticals Inc. seeking next-generation approaches for hemoglobinopathies.
Read More
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.
Read More
Year in review 2024 - Asia deals and innovation

Japan deals big, wins approvals, mulls CGT; Korea, Australia, too

Dec. 31, 2024
By Tamra Sami and Marian (YoonJee) Chu
At Bio Japan 2024, policymakers and industry leaders pledged to make Japan a land of drug discovery to attract native and foreign drug developers. Like many Asian countries, Japan is harnessing and prioritizing the bio sector to drive economic growth, throwing its weight behind its 10-year Bioeconomy initiative to create bioclusters and increase investment.
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Broken chain link

Sangamo stock plunges as Pfizer leaves hemophilia partnership

Dec. 31, 2024
By Lee Landenberger
Sangamo Therapeutics Inc.’s stock sank sharply on the last day of 2025 as Pfizer Inc. handed back the rights to their collaborative gene therapy hemophilia A program. While it was another big loss to Sangamo, which had seen two other major deals fall through in the past two years, the company still has two large collaborations in development.
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