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🚀 Gene Therapy Startups Soar!

Daily news that is actually intellectually stimulating.

Author

Anthony Ao
July 27, 2025

ProKidney’s Rilparencel Shows Strong Phase‑2 Results & Solid Biosciences Gains IND/CTA Approval for SGT‑501

Captured inside a Centers for Disease Control and Prevention (CDC) laboratory, this image depicted a close view of a laboratory technician in the process of pipetting a sample of vitamin E acetate, which would be used in various tests conducted upon a number of electronic cigarettes, referred to as e-cigarettes, or e-cigs, and vaping pens.

Here are two additional biotech‑startup stories that both center on early‑stage gene/cell therapy companies hitting pivotal clinical milestones.

ProKidney’s Cell Therapy Significantly Slows Kidney Disease Progression in Diabetes Patients

ProKidney Corp. announced promising phase-2 results from its regenerative cell therapy, rilparencel (REACT), aimed at treating chronic kidney disease associated with diabetes. The treatment notably slowed kidney function decline by up to 78%, as measured by improvement in estimated glomerular filtration rate (eGFR). Patients receiving two injections showed the greatest benefit, though even those receiving a conditional second dose showed substantial improvement. No serious adverse events were reported among the 49 participants. ProKidney is planning discussions with the FDA to potentially speed up the approval process for its ongoing phase-3 trial.

This news highlights significant progress in regenerative medicine, demonstrating potential for rilparencel to become an effective therapy against diabetic kidney disease—a condition with limited treatment options. Successful progression to phase-3 could pave the way for faster regulatory approval.

Read full article here

Solid Biosciences Gets Green Light for Groundbreaking Gene Therapy Targeting Rare Heart Disorder

Solid Biosciences has received approval from both the U.S. FDA and Health Canada to advance its novel gene therapy, SGT‑501, into clinical trials. This therapy aims to treat catecholaminergic polymorphic ventricular tachycardia (CPVT), a rare, life-threatening heart rhythm disorder with no currently approved treatments. SGT‑501 uses an innovative approach to deliver a corrective gene directly to heart muscle cells, potentially preventing dangerous arrhythmias. With Orphan Drug and Rare Pediatric Disease designations already secured, Solid anticipates beginning a Phase-1b trial by late 2025.

This development highlights the ability of agile biotech companies to quickly bring groundbreaking treatments for ultra-rare diseases from research to clinical testing, potentially offering new hope to patients who previously had no therapeutic options.

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The PhDLevel Team
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