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Home » Authors » Randy Osborne

Randy Osborne

Articles

ARTICLES

Eye and DNA illustration

Series A backers see Ray of hope; $100M for retinal diseases

May 16, 2023
By Randy Osborne
Ray Therapeutics Inc.’s upsized and oversubscribed $100 million series A financing will support the firm’s ongoing efforts with optogenetics, an approach that deploys adeno-associated virus (AAV) gene therapy to deliver a light-sensitive, highly bioengineered protein found in nature to retinal cells.
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IV drip

Sarepta’s DMD gene therapy squeaks past adcom with 8-6 vote

May 12, 2023
By Randy Osborne
The debate over Sarepta Therapeutics Inc.’s gene transfer therapy, SRP-9001 (delandistrogene moxeparvovec), in Duchenne muscular dystrophy (DMD) proved as thorny as expected during a closely watched meeting of the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee. Panelists voted on a single question: “Do the overall considerations of benefit and risk, taking into account the existing uncertainties, support accelerated approval of SRP-9001, using as a surrogate endpoint expression of Sarepta’s microdystrophin at week 12 after administration, for the treatment of ambulatory patients with DMD with a confirmed mutation in the DMD gene?” Balloting turned out 8 yes, 6 no.
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Doctor examining child in wheelchair

Adcom briefing docs mixed; will FDA see Sarepta’s ‘grander vision’ in DMD gene therapy?

May 10, 2023
By Randy Osborne
How grave they might be remains unknown, but regulatory questions have surfaced in briefing documents related to the soon-to-happen panel meeting on Sarepta Therapeutics Inc.’s gene transfer therapy delandistrogene moxeparvovec in Duchenne muscular dystrophy (DMD). The U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee will meet May 12 to discuss the compound, also known as SRP-9001.
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Alpha-galactosidase enzyme

Protalix, Chiesi win US go-ahead for Elfabrio in Fabry disease

May 10, 2023
By Randy Osborne
On the heels of the marketing OK in Europe, Protalix Biotherapeutics Inc. and the Chiesi Group’s global rare diseases unit scored approval of Elfabrio (pegunigalsidase alfa-iwxj) from the U.S. FDA for adults with Fabry disease.
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Protalix, Chiesi win US go-ahead for Elfabrio in Fabry disease

May 10, 2023
By Randy Osborne
Multiple sclerosis

Et tu, Bruton? New approach in MS gains ground despite Merck partial hold

May 9, 2023
By Randy Osborne
Watchers of the Bruton’s kinase (BTK) inhibitor space may be casting renewed skepticism in that direction after Merck KGaA disclosed April 12 that the U.S. FDA placed a partial clinical hold on the sign-up of more patients in work testing evobrutinib in relapsing multiple sclerosis (MS) – but BTK efforts in MS continue in various quarters.
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Hands holding holographic intestine

Crohn’s T-cell mystery in crosshairs of Tscan, Amgen deal; $530M potential

May 9, 2023
By Randy Osborne
Tscan Therapeutics Inc.’s Wall Street-pleasing deal with Amgen Inc. in Crohn’s disease (CD) could expand into ulcerative colitis, but meanwhile is bringing $30 million up front with the potential for more than $500 million in preclinical, clinical, regulatory and commercial milestone payments, plus tiered single-digit royalties. Shares of Waltham, Mass.-based Tscan (NASDAQ:TCRX) closed May 9 at $3.40, up $1.25, or 58%, as the world learned of the multiyear collaboration with Amgen, of Thousand Oaks, Calif., that will use Tscan’s target discovery platform, Targetscan, to identify the antigens recognized by T cells in patients with CD.
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Mirv’n on up: Elahere boosts Immunogen’s economics with phase III win in PROC

May 3, 2023
By Randy Osborne
Immunogen Inc. scored a major win in top-line data from the confirmatory phase III trial called Mirasol evaluating the approved antibody-drug conjugate Elahere (mirvetuximab soravtansine-gynx, “mirv”) vs. chemotherapy in patients with folate receptor alpha-positive platinum-resistant ovarian cancer (PROC). The victory led shares of the Waltham, Mass.-based firm to close May 3 at $12.26, a rise of $7.06, or 135%.
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Kidney disease illustration

Travere to deal with FDA after Filspari door number two opens weak in FSGS Duplex trial

May 2, 2023
By Randy Osborne
Saddled with disappointing results from a phase III trial with Filspari (sparsentan) in focal segmental glomerulosclerosis (FSGS), officials of Travere Therapeutics Inc. stressed the differences between that disorder and IgA nephropathy (IgAN) – for which the drug was cleared by the U.S. FDA in February. Two-year IgAN efficacy data are due in the fourth quarter of this year.
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No letup by Sanofi in Pompe; Maze deal valued as high as $750M

May 1, 2023
By Randy Osborne

A mid-stage prospect for Pompe disease (PD) – this one from Maze Therapeutics Inc. – caught the eye of Sanofi SA, and the pair signed a potential $750 million deal to move along the oral glycogen synthase (GYS1) inhibitor MZE-001, a substrate reduction therapy.


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