Solid Biosciences Receives FDA Rare Pediatric Disease Designation for SGT-212 Dual Route of Administration Gene Therapy for Friedreich’s Ataxia (Globe Newswire)
Solid Biosciences Inc., which acquired UF startup AavantiBio, announced that it received Rare Pediatric Disease designation from the U.S. Food and Drug Administration (FDA) for SGT-212, the Company’s investigational gene therapy for Friedreich’s ataxia (FA).
Gene Therapy Specialist Kriya Raises $313M (Bio Space)
UF startup Kriya is advancing a host of gene therapies for a wide variety of chronic diseases, including geographic atrophy, trigeminal neuralgia and type 1 diabetes.
NIH Award Supports New Extracellular Vesicle Loading Platform To Develop Gene Therapy
UF researcher Mei He, Ph.D., has received a $1.3 million award from the National Institutes of Health to support a new extracellular vesicle platform used to develop gene therapy.
Atsena Therapeutics Announces Oversubscribed $150 Million Series C Financing To Further Advance Ocular Gene Therapy Programs
UF startup Atsena Therapeutics, a clinical-stage gene therapy company focused on reversing or preventing blindness, announced the successful close of an oversubscribed $150 million Series C financing round.
Genascence Announces Six-Month Results From Phase 1b DONATELLO Trial Indicating GNSC-001 Was Safe and Well Tolerated Across Multiple Dosing Arms
UF startup Genascence Corporation, a clinical-stage biotechnology company revolutionizing the treatment of prevalent musculoskeletal diseases with gene therapy, announced positive interim safety and biomarker results from the Phase 1b DONATELLO clinical trial evaluating GNSC-001.
Atsena Therapeutics Announces Dosing Completed in Part A of Phase I/II Clinical Trial Evaluating Gene Therapy ATSN-201 To Treat X-Linked Retinoschisis
UF startup Atsena Therapeutics announced dosing has been completed in Part A of the LIGHTHOUSE study, a Phase I/II clinical trial evaluating subretinal injection of ATSN-201 for the treatment of X-linked retinoschisis (XLRS).
How Atsena Got Its Gene Therapy To Spread Subretinally To Treat XLRS
UF researcher Sanford Boye, co-founder and adviser of UF Startup Atsena Therapeutics, shared insights into how the company developed its gene therapy to achieve subretinal spread for treating XLRS.
Novartis Buys Kate Therapeutics, Eyeing $55B CGT Market Opportunity by 2030
UF Startup Kate Therapeutics, a gene therapy specialist, is being acquired by Novartis in a deal worth up to $1.1 billion. This acquisition aligns with Novartis’s efforts to deliver treatments for neuromuscular disorders and fits into the pharma’s strategy to expand its new modality pipeline, ensuring long-term business sustainability.
Genascence Granted FDA Fast Track Designation for GNSC-001 in Patients With Osteoarthritis (OA) of the Knee
UF Startup Genascence Corporation announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation (FTD) for GNSC-001, a potential first-in-class gene therapy for the treatment of patients with osteoarthritis (OA) of the knee.
Kriya Highlights Positive Preclinical Data for KRIYA-586, a Gene Therapy Product Candidate for Thyroid Eye Disease (TED), at ESOPRS Annual Meeting
UF Startup Kriya Therapeutics, a biopharmaceutical company developing gene therapies, announced preclinical data from its gene therapy program for thyroid eye disease (TED).