Neurogene Reports Interim P-I/II Trial Results on NGN-401 to Treat Pediatric Patients with Rett Syndrome

Shots: Neurogene has reported interim data from the pediatric cohort (4-10yrs.) of the ongoing P-I/II trial assessing NGN-401 for the treatment of females with Rett syndrome As of Oct 30, 2025, all pediatric pts (n=8) had functional gains across Rett syndrome domains, achieving 35 developmental milestones in fine motor, communication & ambulation. These gains were…

ByRidhi RastogiNovember 13, 2025

NEWS

Eli Lilly Strengthens its Gene Therapy Capabilities with Adverum Acquisition

Shots: Eli Lilly has entered into a definitive agreement to acquire Adverum Biotechnologies, incl. its asset Ixo-vec As per the deal, Lilly will acquire Adverum for $3.56/share plus a CVR of ~$8.91 per share, tied to milestones: for ~$1.78 per CVR on Ixo-vec’s FDA approval within 7yrs. of closing & ~$7.13 per CVR upon achieving $1B…

ByRidhi RastogiOctober 27, 2025

NEWS

AviadoBio Enters Exclusive Option & License Agreement with UgeneX Therapeutics to Advance UGX-202

Shots: AviadoBio has entered into an exclusive option & license agreement with UgeneX Therapeutics to develop & commercialize UGX-202 As per the deal, AviadoBio will receive an option to develop & commercialize UGX-202 for retinitis pigmentosa & other indications globally (excl. Greater China), while UgeneX will receive upfront payments, r&d milestones, & sales milestone payments…

ByRidhi RastogiOctober 9, 2025

NEWS

Remedium Bio Joins Forces with Eli Lilly to Advance Gene Therapies for Obesity and Type 2 Diabetes

Shots: Remedium Bio has entered into a multi-target research & development collaboration with Eli Lilly to develop gene therapies targeting multiple targets for the treatment of Obesity & Type 2 Diabetes, leveraging Remedium’s Prometheus platform As per the deal, Remedium will receive an upfront, equity investment as well as development & commercial milestone payments, with tiered…

ByRidhi RastogiSeptember 9, 2025

CXO TALKS

CXO Talks: Rick Modi from Affinia Therapeutics in a Stirring Dialogue Exchange with PharmaShots

Shots: In this episode of the CXO Talks podcast, PharmaShots speaks with Rick Modi, CEO at Affinia Therapeutics Driven by cutting-edge innovation, Affinia is pioneering and re-imagining adeno-associated virus serotype 9 (AAV9), a gene therapy delivery method for conditions affecting cardiac muscle, skeletal muscle, and the central nervous system Rick articulates the diagnostic and therapeutic…

BySaurabh ChaubeySeptember 8, 2025

NEWS

Arrowhead Enters a ~$2.2B Deal with Novartis for ARO-SNCA and TRiM-Based Therapies to Treat Neurodegenerative Diseases

Shots: Arrowhead has granted Novartis exclusive global rights to develop, manufacture, & commercialize ARO-SNCA, which leverages the TRiM platform for SC dosing & targeted gene delivery to treat synucleinopathies, incl. Parkinson’s disease As per the deal, Arrowhead will receive $200M upfront & ~$2B in development, regulatory, and sales milestones, with tiered royalties on sales up…

ByRidhi RastogiSeptember 3, 2025

NEWS

Nuevocor Reports the US FDA’s IND Clearance of NVC-001 for LMNA DCM

Shots: The US FDA has approved the IND application of NVC-001 to treat LMNA-related dilated cardiomyopathy (LMNA DCM), enabling a P-I/II trial to start in early 2026 Preclinical studies showed that NVC-001 significantly improved survival and cardiac function. They planned a P-I/II trial, a 52-week, open-label, multicenter ascending-dose study to assess the safety, tolerability, and…

ByPrince GiriJune 11, 2025

NEWS

Chengdu Origen and Vanotech Report the First Patient Dosing in P-I (VAN-2401) Trial of KH658 for Wet Age-related Macular Degeneration

Shots: Chengdu Origen & Vanotech has reported the first pts dosing in Vanotech-led P-I (VAN-2401) trial assessing KH658 for wet age-related macular degeneration (wet AMD) in the US The P-I trial will assess safety, tolerability, & efficacy of single suprachoroidal KH658 gene therapy in ~9 previously treated wet AMD pts that are responsive to anti-VEGF therapy…

ByRidhi RastogiMay 5, 2025

NEWS

Abeona Therapeutics’ Zevaskyn Receives the US FDA Approval for Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Shots: The US FDA has approved Zevaskyn (prademagene zamikeracel) for treating wounds in adult & pediatric RDEB pts based on the intra-pts controlled P-III (VIITAL) trial & has also granted RPD PRV to Abeona, which the company intends to monetize; Zevaskyn to be commercially available in Q3’25 The P-III Trial assessed Zevaskyn in RDEB pts,…

ByRidhi RastogiApril 30, 2025

NEWS

SineuGene Therapeutics Reports the US FDA’s IND Clearance of SNUG01 for Amyotrophic Lateral Sclerosis

Shots: The US FDA has granted IND clearance to SNUG01 for ALS SNUG01 will be evaluated in a P-I/IIa dose-escalation & expansion trial to assess its safety, tolerability, & preliminary efficacy in ALS adults; SineuGene to partner with academics & institutions to lead clinical development of SNUG01 SNUG01, a TRIM72-targeted gene therapy, counteracts ALS through…

ByRidhi RastogiApril 3, 2025

Neurogene Reports Interim P-I/II Trial Results on NGN-401 to Treat Pediatric Patients with Rett Syndrome

AviadoBio Enters Exclusive Option & License Agreement with UgeneX Therapeutics to Advance UGX-202

Remedium Bio Joins Forces with Eli Lilly to Advance Gene Therapies for Obesity and Type 2 Diabetes

CXO Talks: Rick Modi from Affinia Therapeutics in a Stirring Dialogue Exchange with PharmaShots

Arrowhead Enters a ~$2.2B Deal with Novartis for ARO-SNCA and TRiM-Based Therapies to Treat Neurodegenerative Diseases

Nuevocor Reports the US FDA’s IND Clearance of NVC-001 for LMNA DCM

Abeona Therapeutics’ Zevaskyn Receives the US FDA Approval for Recessive Dystrophic Epidermolysis Bullosa (RDEB)

SineuGene Therapeutics Reports the US FDA’s IND Clearance of SNUG01 for Amyotrophic Lateral Sclerosis

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