This Founder’s Gene Therapy Company Helped Blind Kids See Again https://t.co/o7xS8Kj7vL https://t.co/PoJUTjfJXf ...

Core Insights - Krystal Biotech received FDA approval to update the label of Vyjuvek to include dystrophic epidermolysis bullosa (DEB) patients from birth, leading to an 8.4% increase in share price following the announcement [1][7]. Product Update - The label update allows for greater flexibility in the use of Vyjuvek, enabling patients and caregivers to administer the treatment at home and manage wound dressings more conveniently [2][7]. - The FDA's decision was based on data from an open-label extension study and real-world experience, confirming the long-term safety and efficacy of Vyjuvek across all ages [2]. Market Context - Vyjuvek was previously approved in 2023 as the first revocable gene therapy for DEB patients aged six months or older, a rare and severe genetic condition caused by mutations in the COL7A1 gene [3]. - Year-to-date, Krystal Biotech's shares have decreased by 0.7%, while the industry has seen a 3.6% increase [4]. Sales Performance - Vyjuvek recorded sales of $184.2 million in the first half of 2025, with expectations for further sales growth due to the recent label update and approvals in Europe and Japan [10]. - The company has secured over 575 reimbursement approvals for Vyjuvek in the U.S. since its launch, indicating steady progress in market access [8]. Expansion Plans - Krystal Biotech plans to launch Vyjuvek in Germany in Q3 and in France in Q4 of 2025, following its approval in Japan for DEB patients from birth [9].

Core Insights - The U.S. FDA approved Krystal Biotech's label update for Vyjuvek, expanding its eligible patient population to include dystrophic epidermolysis bullosa (DEB) patients from birth and allowing greater flexibility in wound dressing management [1][3][4] Group 1: Product Update - The label update for Vyjuvek allows for the treatment of DEB patients from birth, enhancing the product's market potential [1] - Patients can now remove wound dressings during the next dressing change instead of waiting 24 hours, integrating Vyjuvek into existing wound care routines [3] Group 2: Clinical Data and Safety - The label update is supported by real-world data collected since Vyjuvek's U.S. launch and results from an open label extension study, confirming the long-term safety and efficacy of Vyjuvek across all ages [4] Group 3: Market Reaction - Wall Street responded positively to the broader label, with analysts noting Vyjuvek's at-home dosing appeal and its potential to improve patient quality of life, which could lead to strong revenue growth [5] - KRYS stock increased by 6.01% to $152.32 following the announcement [6]

Group 1 - Krystal Biotech announced FDA approval for an updated label for its gene therapy, Vyjuvek (beremagene geperpavec) [6] - The new approval expands patient eligibility to include individuals with dystrophic epidermolysis bullosa from birth [6] - The updated label allows for broader access to the therapy for affected patients [6]

SANTA ANA, Calif.--(BUSINESS WIRE)--FUJIFILM Biosciences, a global leader in the innovation and manufacture of cell culture solutions for the Life Science market, today announced the commercial launch of BalanCD HEK293 Perfusion A. Designed to expand and augment the company's portfolio of gene therapy application solutions, BalanCD HEK293 Perfusion A uses suspension HEK293 cells and perfusion technology to provide reliable, efficient, and scalable production of viral vectors for development of. ...

Group 1: Company Overview - Sana Biotechnology (SANA) is valued at $837.6 million and focuses on creating modified cells and gene therapies to repair or replace damaged cells and control gene expression [4] - The company has significantly expanded its financial runway, ending Q2 with $72.7 million in cash, which increased to a pro forma $177.2 million after capital raises, expected to fund operations until the second half of 2026 [1] Group 2: Product Development and Clinical Trials - Sana is developing a functional treatment for diabetes that does not require lifelong immunosuppression, which is considered a promising initiative [1] - The company is also working on allogeneic CAR T therapies, including SC291 for autoimmune diseases and SC262 for relapsed/refractory B-cell malignancies, with results from Phase 1 trials expected by 2025 [2] - The UP421 trial, using hypoimmune-modified pancreatic islet cells for type 1 diabetes, has shown success, leading to a 100% increase in stock price year-to-date [3] Group 3: Market Sentiment and Analyst Ratings - Wall Street rates SANA stock as a "Strong Buy," with seven out of nine analysts recommending it, and an average target price of $9.17, indicating a potential upside of 169.7% [6] - The highest estimate for the stock is $15, suggesting a possible increase of 341.2% in the next 12 months [6]

As filed with the Securities and Exchange Commission on September 12, 2025. Registration Statement No. 333- UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM S-1 REGISTRATION STATEMENT UNDER THE SECURITIES ACT OF 1933 GENPREX, INC. (Exact name of registrant as specified in its charter) (State or other jurisdiction of incorporation or organization) (Primary Standard Industrial Classification Code Number) Delaware 2834 90-0772347 (I.R.S. Employer Identification Number) 3300 Bee Cave ...

Core Insights - Sanofi's investigational gene therapy SAR402663 received fast track designation from the FDA for treating neovascular (wet) age-related macular degeneration (AMD) [1][6] Group 1: Fast Track Designation Benefits - Fast track designation aims to expedite the development and review of drugs addressing serious conditions and unmet medical needs, allowing for earlier patient access [2] - The designation enables rolling review, allowing Sanofi to submit completed sections of the regulatory filing for SAR402663 as they become available, potentially speeding up the evaluation process [2][6] Group 2: Product Details - SAR402663 is a one-time intravitreal gene therapy targeting vascular endothelial growth factor (VEGF), which is responsible for abnormal blood vessel growth in the eye [5][6] - The therapy aims to reduce treatment burden by eliminating the need for frequent intravitreal injections [5][6] Group 3: Clinical Development - Sanofi is currently conducting a phase I/II study for SAR402663 in patients with neovascular/wet AMD, a severe condition affecting over a million people in the United States [7] Group 4: Competitive Landscape - Key competitors include AbbVie's ABBV-RGX-314, which is also a one-shot gene therapy targeting VEGF and is in pivotal studies for wet AMD, with data expected next year [8] - Another competitor is 4D Molecular Therapeutics' 4D-150, which is also targeting VEGF and is undergoing late-stage studies for wet AMD, with data anticipated in the second half of 2027 [9]

Core Insights - The FDA has granted fast track designation to Sanofi's SAR402663, a one-time intravitreal gene therapy for neovascular age-related macular degeneration (AMD) [1][7] - This designation aims to expedite the development and review of treatments for serious conditions, addressing unmet medical needs [1] Company Overview - Sanofi is an R&D driven biopharma company focused on improving lives through innovative medicines and vaccines, with a commitment to addressing urgent healthcare challenges [5] - The company is exploring therapies for various neurological diseases, including AMD, and aims to leverage scientific innovation in ophthalmology for growth [4] Product Details - SAR402663 delivers genetic material that encodes soluble FLT01, designed to inhibit vascular endothelial growth factor (VEGF), addressing the underlying pathology of neovascular AMD [2] - The therapy aims to reduce treatment burden by eliminating the need for frequent intravitreal injections, which is a significant advantage for patients [2][7] Market Context - Neovascular AMD affects over one million people in the US and approximately six million people worldwide, significantly impacting quality of life [3] - The condition is characterized by abnormal blood vessel growth beneath the retina, leading to vision loss and potential blindness [3]

Core Insights - The FDA has granted fast track designation to Sanofi's SAR402663, a one-time intravitreal gene therapy for neovascular age-related macular degeneration (AMD) [1][7] - The fast track designation aims to expedite the development and review of treatments for serious conditions, addressing unmet medical needs [1] Company Overview - Sanofi is an R&D driven biopharma company focused on improving lives through innovative medicines and vaccines, with a commitment to addressing urgent healthcare challenges [5] - The company is exploring therapies for various neurological diseases, including AMD, and aims to leverage scientific innovation in ophthalmology for growth [4] Product Details - SAR402663 delivers genetic material designed to inhibit vascular endothelial growth factor (VEGF), addressing the underlying pathology of neovascular AMD by reducing abnormal blood vessel growth and minimizing retina damage [2] - The therapy aims to significantly reduce the treatment burden by eliminating the need for frequent intravitreal injections [2] Market Context - Neovascular AMD affects over one million people in the US and approximately six million people worldwide, leading to significant vision loss and impacting quality of life [3][7] - AMD is a progressive degeneration of the retina that affects around 200 million people globally, highlighting the substantial market potential for effective treatments [3]