Musculoskeletal

Nucleotide’s in: Gene therapy high water lifts Affinia with $60M series A

April 1, 2020

The allure of gene therapy was proved yet again as Waltham, Mass.-based Affinia Therapeutics Inc. bagged an oversubscribed $60 million series A financing to boost the push for drugs to benefit people affected by muscle and central nervous system conditions.

Design Therapeutics raises $45M to take on degenerative disorders

March 20, 2020

By Michael Fitzhugh

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Design Therapeutics Inc., a San Diego startup developing new therapies for degenerative disorders caused by nucleotide repeat expansions, has raised $45 million in series A financing.

Archimedes entreaties: Needful FSHD world to move via Fulcrum bid with losmapimod?

March 10, 2020

By Randy Osborne

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With a phase IIb readout coming in the third quarter of 2020, Cambridge, Mass.-based Fulcrum Therapeutics Inc. might be set up for a win in facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder for which there’s no treatment.

Keros’ pedal-down TGF-beta push touts safety, draws $56M series C

March 5, 2020

By Randy Osborne

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Keros Therapeutics Inc. CEO Jasbir Seehra told BioWorld that he plans to use at his new company lessons learned as co-founder of Acceleron Pharma Inc., where work with receptors in the TGF-beta superfamily “taught me the potential of the biology and those molecules, but also the limitations” with regard to safety that need to be surmounted.

Trikafta leads the charge for Vertex’s strong 2019

January 31, 2020

By Lee Landenberger

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Vertex Pharmaceuticals Inc. brought in $4.16 billion in product revenues in 2019, bolstered in large part by the late October approval of Trikafta, earning $420 million in the few weeks that remained in the year.

Blueprint for a backup?

Ipsen puts bone disorder drug dosing on ice after futility analysis

January 24, 2020

By Michael Fitzhugh

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Expectations that a phase III trial of Ipsen SA's palovarotene will miss its primary endpoint of reducing abnormal bone growth among people with a rare bone disorder led the company to pause dosing in that study and another as it evaluates next steps.

Transcenta closes $100M series B+ to advance pipeline and IPO plan

January 10, 2020

By Elise Mak

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BEIJING – China-U.S. biotech Transcenta Holding Ltd., headquartered in Shanghai and Boston, completed a series B+ financing round to secure $100 million to continue its efforts in developing oncology and bone disorder drugs and to prepare for an IPO.

Australia’s Antisense Therapeutics’ phase II DMD trial meets primary endpoints for disease progression

December 31, 2019

By Tamra Sami

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PERTH, Australia – On the heels of a $1.15 billion deal between Roche Holding AG and Sarepta Therapeutics Inc. for Sarepta’s gene therapy to treat Duchenne muscular dystrophy (DMD), another therapy to treat DMD is emerging from Down Under.

High-profile failure hits Stealth's phase III mitochondrial myopathy trial

December 20, 2019

By Michael Fitzhugh

No Comments

A medicine Stealth Biotherapeutics Inc. has been developing for the potential treatment of primary mitochondrial myopathy (PMM) missed the co-primary endpoints of a key phase III trial, sending company shares (NASDAQ:MITO) down 65.9% to close at $4.75 on Friday.

FDA’s change of heart

Sarepta’s Vyondys 53 wins a surprise accelerated approval

December 13, 2019

By Lee Landenberger

No Comments

In August the FDA was skeptical about Sarepta Therapeutics Inc.’s injectable Vyondys 53 (golodirsen), but that changed swiftly Friday with the agency’s accelerated approval for the Duchenne muscular dystrophy (DMD) follow-on therapy, the first treatment specifically for this subtype.

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Nucleotide’s in: Gene therapy high water lifts Affinia with $60M series A

Design Therapeutics raises $45M to take on degenerative disorders

Archimedes entreaties: Needful FSHD world to move via Fulcrum bid with losmapimod?

Keros’ pedal-down TGF-beta push touts safety, draws $56M series C

Trikafta leads the charge for Vertex’s strong 2019

Ipsen puts bone disorder drug dosing on ice after futility analysis

Transcenta closes $100M series B+ to advance pipeline and IPO plan

Australia’s Antisense Therapeutics’ phase II DMD trial meets primary endpoints for disease progression

High-profile failure hits Stealth's phase III mitochondrial myopathy trial

Sarepta’s Vyondys 53 wins a surprise accelerated approval

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