Core Insights - Pacira BioSciences, Inc. is presenting new data on its gene therapy candidate PCRX-201 at the 2025 EULAR Annual Congress, highlighting its commitment to innovative, non-opioid pain therapies [1] Group 1: Clinical Data and Study Results - The Phase 1 study of PCRX-201 demonstrated that a single intra-articular injection was safe and provided sustained clinical benefits for patients with moderate-to-severe knee osteoarthritis over three years [2] - In a previous Phase 1 study, PCRX-201 showed sustained improvements in knee pain, stiffness, and function for two years post-administration, with a well-tolerated safety profile [3] - PCRX-201 has received RMAT designation from the FDA and ATMP designation from the EMA, marking it as the first gene therapy to achieve these clinical results in knee osteoarthritis [3] Group 2: Ongoing Research and Development - Following promising Phase 1 results, dosing is currently underway in a Phase 2 study (ASCEND study) for the treatment of knee osteoarthritis [4] - The company is advancing the development of PCRX-201 as a novel, locally administered gene therapy aimed at treating prevalent diseases like osteoarthritis [5] Group 3: Company Overview - Pacira BioSciences focuses on delivering innovative, non-opioid pain therapies, with three commercial-stage products: EXPAREL, ZILRETTA, and iovera® [5] - EXPAREL is a long-acting local analgesic approved for various postsurgical pain management applications, while ZILRETTA is an extended-release injection for osteoarthritis knee pain [5]

Second ophthalmic program in clinic - KB801 for the treatment of neurotrophic keratitis Upcoming clinical readouts in 2025 - CF, AATD, ocular complications of DEB, and aesthetics Strong balance sheet, ending the quarter with $765.3 million in cash and investments VYJUVEK approved in Europe for the treatment of DEB patients from birth $88.2 million in 1Q VYJUVEK revenue and $429.4 million since launch in Q3 2023 PITTSBURGH, May 06, 2025 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the "Company") (NASDAQ: KRYS) ...

Core Insights - uniQure N.V. will report its first quarter 2025 financial results on May 9, 2025, before market open, and will host an earnings call at 8:30 a.m. ET to provide updates on its pipeline and AMT-130 [1] - The company is initiating quarterly earnings calls to enhance engagement with the investment community as it progresses towards a BLA submission and potential commercialization [1] Company Overview - uniQure is a leading gene therapy company focused on delivering transformative therapies for patients with severe medical needs, including a gene therapy for hemophilia B, which marks a significant milestone in genomic medicine [3] - The company is advancing a pipeline of proprietary gene therapies targeting Huntington's disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and other severe diseases [3]

Improvements in subjective and objective measures of efficacy observed at six months persisted for one year in patients with severe vision impairment from inherited retinal degeneration due to mutations in the LCA5 gene Administration of OPGx-LCA5 by subretinal injection was well tolerated by study participants RESEARCH TRIANGLE PARK, N.C., May 05, 2025 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage ophthalmic biotechnology company developing gene therapies for the treatment of inhe ...

Core Points - Adverum Biotechnologies granted inducement awards to seven new employees, including non-qualified stock options for 32,620 shares and restricted stock units (RSUs) for 16,310 shares under its 2017 Inducement Plan [1][2] - The stock options have an exercise price of $3.33 per share, equal to the closing price on the grant date, and vest over four years [2] - The RSUs vest over three years, with one-third vesting on each anniversary of the applicable vesting commencement date [2] Company Overview - Adverum Biotechnologies is a clinical-stage company focused on gene therapy to preserve sight in prevalent ocular diseases, aiming to develop functional cures to restore vision and prevent blindness [3] - The company utilizes its proprietary intravitreal (IVT) platform to create durable, single-administration therapies, reducing the need for frequent ocular injections [3] - Adverum is evaluating its gene therapy candidate, ixoberogene soroparvovec (Ixo-vec), as a one-time IVT injection for patients with neovascular or wet age-related macular degeneration [3]

Core Insights - Pacira BioSciences is set to present findings from its Phase 1 study of PCRX-201, a gene therapy for knee osteoarthritis, at the ASGCT Annual Meeting on May 15, focusing on the impact of neutralizing antibodies on therapy efficacy and safety [1][2] - The company has reported promising Phase 1 results showing sustained improvements in knee pain and function over two years, leading to regulatory designations from the FDA and EMA [4][5] Company Overview - Pacira BioSciences specializes in innovative, non-opioid pain therapies, with three commercial-stage products: EXPAREL, ZILRETTA, and iovera, alongside the development of PCRX-201 [6] - PCRX-201 utilizes a proprietary high-capacity adenovirus vector platform and targets chronic inflammatory processes in knee osteoarthritis, a condition affecting over 14 million individuals in the U.S. [3][4] Upcoming Events - The presentation titled "Understanding the Clinical Immunogenicity of Locally Injected HCAd Vector Provides Insight into Optimizing Dosing Strategy" will be delivered by Dr. MiJeong Kim on May 15 [2] - A symposium on "High-Capacity Adenoviral Vectors: Advancing Gene Therapy Beyond AAV to Deliver Cost-Effective Therapies for Common Diseases" is scheduled for May 16 [3]

Core Viewpoint - Genprex, Inc. announced positive preclinical data for Reqorsa® Gene Therapy, demonstrating its robust anti-tumor effects in KRASG12C mutant non-small cell lung cancer (NSCLC), both alone and in combination with LUMAKRAS® [1][2]. Group 1: Preclinical Data Presentation - The positive preclinical data was presented at the 2025 American Association for Cancer Research (AACR) Annual Meeting, highlighting the therapeutic potential of REQORSA in treating Ras inhibitor resistant lung cancer [1][2]. - The poster titled "Overcoming sotorasib acquired resistance in KRASG12C mutant NSCLC by TUSC2 gene therapy" showcased that TUSC2 gene therapy effectively overcomes acquired resistance to sotorasib in mouse xenografts [3]. Group 2: Efficacy of Reqorsa - TUSC2 transfection significantly reduced colony formation and increased apoptosis in acquired resistance cell lines, indicating strong anti-tumor efficacy [3]. - REQORSA alone showed a strong anti-tumor effect on TC314AR PDXs, while sotorasib alone exhibited no significant activity [3]. - A synergistic effect was observed when combining REQORSA with sotorasib, enhancing tumor control compared to either treatment alone [3]. Group 3: Technology and Delivery System - Reqorsa consists of a plasmid containing the TUSC2 gene encapsulated in non-viral lipid-based nanoparticles, specifically targeting cancer cells while minimizing uptake by normal tissue [5]. - Laboratory studies indicated that the uptake of TUSC2 in tumor cells after REQORSA treatment was 10 to 33 times higher than in normal cells [5]. Group 4: Company Overview - Genprex, Inc. is a clinical-stage gene therapy company focused on developing therapies for cancer and diabetes, utilizing its Oncoprex® Delivery System for gene administration [6][7]. - The company is advancing its pipeline of gene therapies and has received Fast Track Designation from the FDA for its lung cancer clinical programs [7].

Core Viewpoint - Genprex, Inc. has announced the presentation of positive preclinical data for its diabetes gene therapy candidate GPX-002 at the upcoming ASGCT Annual Meeting, highlighting the potential of gene therapy as a curative treatment for diabetes [1][2]. Group 1: Research and Development - The oral presentation will focus on the immune modulation and reprogramming of alpha cells to beta-like cells in a diabetic non-human primate model, showcasing the effectiveness of the rAAV gene therapy approach [3]. - The study demonstrated that retrograde intraductal infusion of rAAV6 can convert alpha cells into insulin-secreting beta-like cells, reversing diabetes in mouse models [4]. - One month post-infusion, non-human primates exhibited improved glucose tolerance and reduced insulin requirements, indicating the therapy's durable effects [5]. Group 2: Immune Response Management - The research evaluated the immune response to direct infusion of rAAV into the pancreatic duct and how to manage potential anti-viral immunity, which can hinder gene therapy success [4][5]. - Temporary immunosuppression using a combination of rituximab, rapamycin, and steroids was largely effective in preventing anti-viral immune responses, allowing sustained therapeutic effects [6]. Group 3: Product Overview - GPX-002 is being developed for both Type 1 and Type 2 diabetes, utilizing an AAV vector to deliver Pdx1 and MafA genes directly into the pancreatic duct [7]. - In Type 1 diabetes, GPX-002 aims to transform alpha cells into functional beta-like cells, while in Type 2 diabetes, it is believed to rejuvenate exhausted beta cells [7]. Group 4: Company Background - Genprex, Inc. is a clinical-stage gene therapy company focused on developing therapies for cancer and diabetes, collaborating with leading institutions to advance its drug candidates [8].

Core Insights - The cone rod dystrophy market is projected to grow from US$131.29 million in 2024 to US$177.59 million by 2030, driven by increased awareness and demand for effective treatments [2][3] - The market is expected to experience a compound annual growth rate (CAGR) of 5.33% from 2025 to 2030 [3] Market Segmentation Analysis - X-Linked Cone Rod Dystrophy is the leading type in the market, with ongoing research into gene therapies and neuroprotective agents [4] - Gene therapy holds the highest market share in treatment options, particularly through adeno-associated virus vectors [4] - Hospitals dominate the end user segment, benefiting from advanced treatment options and expanding infrastructure, especially in emerging markets [5] Regional Insights - North America leads the market due to high prevalence of eye disorders and technological advancements [6] - Asia Pacific is the fastest-growing region, with significant contributions from China's healthcare investments and Japan's market share [6] Market Dynamics - Growth drivers include rising incidence of cone rod dystrophy, increased R&D activities, and advancements in gene therapy [7] - Challenges include a limited patient pool and inconsistency in insurance coverage [7] - Trends such as early detection and personalized medicine are improving the market environment for new therapies [7] Competitive Landscape - Key players include SparingVision, Beacon Therapeutics, and Nanoscope Therapeutics, with mergers and acquisitions shaping the market [8] - Notable product SPVN06 from SparingVision is undergoing trials for various genetic mutations associated with Retinitis Pigmentosa [8]

Company Overview - Insmed Incorporated is a global biopharmaceutical company focused on delivering first- and best-in-class therapies for patients with serious diseases [3][4] - The company is advancing a diverse portfolio of approved and investigational medicines, particularly in pulmonary and inflammatory conditions [3] - Insmed's advanced programs include a therapy approved in the United States, Europe, and Japan for a chronic lung disease [3] Upcoming Event - Management will present at the BofA Securities 2025 Health Care Conference on May 13, 2025, at 2:20 p.m. PT [1] - The event will be webcast live and available for 30 days post-event on the company's investor relations website [2] Company Recognition - Insmed has been recognized as one of the best employers in the biopharmaceutical industry, including being named the No. 1 Science Top Employer for four consecutive years [4]