Financial Data and Key Metrics Changes - REGENXBIO ended the quarter on December 31, 2024, with cash, cash equivalents, and marketable securities of $245 million, down from $314 million as of December 31, 2023, primarily due to cash used for operating activities [26] - R&D expenses were $209 million for the year ended December 31, 2024, compared to $232 million in 2023, attributed to decreases in headcount and preclinical activities [26][27] - The company expects the cash runway to fund operations into the second half of 2026, with potential extensions through non-dilutive financing options [27] Business Line Data and Key Metrics Changes - The pivotal study for RGX-202 is advancing rapidly, with nearly half of the enrollment completed, and the company expects to submit a BLA under the accelerated approval pathway by mid-2026 [12][29] - The BLA for RGX-121 has been submitted, with potential FDA approval anticipated in the second half of 2025 [7][24] - The diabetic retinopathy program is moving towards a pivotal program with AbbVie, with successful end-of-Phase II meetings held with the FDA [14][20] Market Data and Key Metrics Changes - The partnership with Nippon Shinyaku for MPS programs is strategically significant, combining REGENXBIO's development expertise with Nippon's commercialization experience [9][10] - The company is preparing for potential approval of RGX-121 for MPS II in Q4 2025, which could enhance its market position [10][24] Company Strategy and Development Direction - REGENXBIO aims to launch multiple first or best-in-class gene therapies, focusing on sustainable profitability and leveraging over 15 years of gene therapy leadership [8][29] - The company is positioned to deliver on multiple late-stage opportunities, with a focus on differentiation against standard care and available treatments [29][31] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong momentum across the pipeline, with key milestones approaching [29] - The company believes it is well-positioned to deliver transformative treatments for patients in need of new options, particularly in the Duchenne muscular dystrophy and retinal disease markets [29][31] Other Important Information - The company has a robust commercial capability and global partnerships, which are expected to support the launch of its gene therapies [7][10] - REGENXBIO is actively working on non-dilutive financing options, including milestone payments and potential monetization of a priority review voucher [27] Q&A Session Summary Question: Can you delve deeper into the components of non-dilutive financing? - Management outlined three components: expected milestone payments from the diabetic retinopathy program, potential monetization of a priority review voucher upon regulatory approval, and the reversion of royalty income from Zolgensma once a cap is reached [36][39][40] Question: What are the expectations around potentially going to an advisory committee for RGX-202? - Management indicated that while they do not foresee a significant issue that would necessitate an advisory committee, they are prepared for it if required [46][47] Question: Can you provide an update on the NDA and patient data for the one to three-year-old cohort? - Management plans to update on one patient from the one to four cohort at the upcoming MDA meeting, with expectations of higher microdystrophin levels in younger patients [51][53] Question: What is the current pace of enrollment for the DMD pivotal trial? - Management reported encouraging enrollment and expects acceleration as more sites are activated, with a significant number of patients showing interest [58][59] Question: Are cardiac endpoints being measured in the DMD study? - Management confirmed that they are measuring cardiac endpoints, including ejection fraction and troponin levels, but noted that significant changes may not be observable in younger patients [67][72] Question: Will enrollment for the wet AMD study be cut short to accelerate timelines? - Management stated that they will not cut enrollment short, as they aim to fully characterize safety and efficacy in collaboration with AbbVie [75][76]

Company Overview - Fractyl Health is developing therapies to address the \$250 billion untapped market in weight maintenance[5,8] - The company is developing Revita, an outpatient endoscopic procedure, and Rejuva, a one-time pancreatic gene therapy platform for T2D and obesity[5] - Key clinical milestones are expected in 2025[5] Revita Program - Revita is a procedural therapy targeting gut dysfunction, a root cause of obesity and T2D[5,12,13] - Pooled data from Revita clinical studies demonstrate durable weight maintenance[9] - The REMAIN-1 pivotal study midpoint data is expected in Q2 2025, with full study enrollment in summer 2025[9] - Only 14% of patients are interested in weight loss drugs if informed about the risk of weight regain after stopping them[21] Rejuva Program - Rejuva is a pancreatic gene therapy platform with potential for one-time treatment of T2D and obesity[5,46] - The company plans to submit the first Clinical Trial Application (CTA) module for RJVA-001 in H1 2025 and expects preliminary data in 2026, assuming CTA authorization[9,48,73] - Preclinical data shows RJVA-001 efficacy in db/db and DIO mouse models of T2D and obesity is superior to chronic semaglutide[73] - Local delivery of Rejuva enables low viral genome dosing with limited systemic virus exposure[54]

Core Viewpoint - Krystal Biotech's Vyjuvek has received a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) for the treatment of dystrophic epidermolysis bullosa (DEB), with a final decision expected in Q2 2025 [1][3]. Group 1: Product and Approval Details - Vyjuvek is a non-invasive, topical gene therapy designed to deliver two copies of the COL7A1 gene directly to DEB wounds [2]. - The CHMP's favorable opinion supports administration in healthcare settings or at home, with trained patients or caregivers able to apply it themselves [3]. - The positive opinion is based on results from phase I/II GEM-1 and phase III GEM-3 studies, demonstrating successful gene delivery and durable wound closure [5]. Group 2: Market and Financial Performance - Krystal Biotech's shares have declined 18.3% over the past three months, contrasting with a 0.6% decline in the industry [4]. - In Q4 2024, the company reported total revenues of $91.1 million, a 116% increase year-over-year, solely from Vyjuvek sales [8]. - As of February 2025, the company secured over 510 reimbursement approvals for Vyjuvek in the U.S., with a gross margin of 95% in Q4 2024 and high patient adherence at 85% [9]. Group 3: Future Launch Plans - Krystal Biotech plans to launch Vyjuvek for DEB in Germany around mid-2025, with additional launches in France and other EU countries later in 2025 [7].

As filed with the Securities and Exchange Commission on April 29, 2024. Registration No. 333-278006 UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 Amendment No. 3 to FORM S-1 REGISTRATION STATEMENT UNDER THE SECURITIES ACT OF 1933 MUSTANG BIO, INC. (Exact name of registrant as specified in its charter) Delaware (State or other jurisdiction of incorporation or organization) 2836 (Primary Standard Industrial Classification Code Number) 47-3828760 (I.R.S. Employer Identification Number ...

MUSTANG BIO, INC. (Exact name of registrant as specified in its charter) Delaware (State or other jurisdiction of incorporation or organization) As filed with the Securities and Exchange Commission on March 15, 2024. Registration No. 333- UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM S-1 REGISTRATION STATEMENT UNDER THE SECURITIES ACT OF 1933 2836 (Primary Standard Industrial Classification Code Number) 47-3828760 (I.R.S. Employer Identification Number) 377 Plantation Street W ...

Table of Contents As filed with the Securities and Exchange Commission on July 7, 2023 Registration No. 333- UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM S-1 REGISTRATION STATEMENT UNDER THE SECURITIES ACT OF 1933 Benitec Biopharma Inc. (Exact name of registrant as specified in its charter) Delaware 2834 84-462-0206 (State or other jurisdiction of incorporation or organization) (Primary Standard Industrial Classification Code Number) (I.R.S. Employer Identification Number) Dr ...