Summary of Absci's Healthcare Innovation Conference Call Company Overview - **Company**: Absci - **Industry**: Biopharmaceuticals, specifically focusing on generative AI drug creation and antibody development - **Key Personnel**: Sean McClain (CEO), Zach Johansson (CFO and CBO) [1][2] Core Technology and Strategy - Absci originally focused on protein-protein interactions and has evolved to utilize generative AI for drug creation, particularly antibodies [3][4] - The company aims to target hard-to-drug biological targets, such as GPCRs and ion channels, leveraging their technology to create first-in-class and best-in-class assets [5] Lead Programs ABS-201 - **Target**: Prolactin receptor - **Indications**: - Androgenic alopecia (common baldness affecting 80 million Americans) - Endometriosis (to be discussed in future updates) [6][7] - **Mechanism**: Blocking the prolactin receptor can transition hair follicles from a catagen state (apoptosis) to an active growth state, showing superior efficacy compared to minoxidil in preclinical studies [6][10] - **Clinical Timeline**: - First participant dosing expected in December 2023 - Proof of concept readout anticipated in the second half of 2024 [6][13] Market Opportunity - The hair regrowth market is seen as a significant opportunity, with patients willing to pay out of pocket for effective treatments [7][10] - Current treatments like minoxidil and finasteride have limitations, including side effects and lack of condition modification [10][11] - The proposed treatment could provide long-lasting results with just two to three injections, potentially offering benefits for up to three years [11][17] Competitive Landscape - The hair regrowth market has seen little innovation, primarily dominated by finasteride and minoxidil [10] - Absci's approach is condition-modifying, which could differentiate it from existing treatments [11] - The company plans to engage both physicians and direct-to-consumer strategies for commercialization [17][18] Financial Position - As of Q2, Absci had approximately $117.5 million in cash and equivalents, with an additional $64 million raised in July 2023 [42][43] - The current financial runway extends into the first half of 2028, allowing for the advancement of multiple programs [42] Upcoming Catalysts - Key upcoming events include: - Large pharma partnership announcement in the near term - Entry into the clinic with ABS-201 in December 2023 - Interim readout on the alopecia trial in the second half of 2024 [44] Additional Insights - The company is exploring other indications and potential partnerships, particularly in inflammatory bowel disease (IBD) [31][32] - There is a focus on safety and efficacy, with ongoing discussions about the mechanism of action and potential side effects of blocking the prolactin receptor [23][24] Conclusion Absci is positioned to make significant advancements in the biopharmaceutical industry with its innovative approach to drug development, particularly in the hair regrowth market. The upcoming clinical trials and potential partnerships could enhance its market presence and financial stability.

Summary of McKesson's 2025 Conference Call Company Overview - **Company**: McKesson Corporation (NYSE: MCK) - **Event**: UBS Healthcare Conference - **Date**: November 10, 2025 Key Points Industry Insights - **North American Pharmaceutical Business**: McKesson has resegmented its business to provide clarity on strategies, separating specialty oncology from the North American Pharmaceutical segment, which includes the US Pharma and Canadian distribution businesses [3][5][6] - **Market Trends**: The company has observed consistent growth in utilization, driven by demographic trends and innovations in drug development and distribution [7][9] - **Specialty Drugs**: There is a notable shift towards specialty drugs, which are becoming a larger part of the business mix, contributing to improved margins [9][10] Financial Performance - **Operating Expense Leverage**: Over the past five years, McKesson has improved its operating expense leverage by over 1,000 basis points, attributed to investments in automation and technology [17][18] - **Revenue Growth**: The adjusted EPS guidance for the year is between $38.35 and $38.85, representing a growth of 16%-18%, or 18%-20% when excluding prior year gains [63] Strategic Initiatives - **Automation and AI**: McKesson is investing in automation and AI to enhance operational efficiency, with a new distribution center in Ohio being 90% automated [16][18] - **Oncology and Specialty Services**: The company is focused on expanding its oncology services and exploring opportunities in other specialties, particularly in high drug spend areas like retina and ophthalmology [35][44] Pricing and Market Dynamics - **Generic Pricing**: The pricing environment for generics has been stable, with month-to-month variations not significantly impacting long-term strategies [21][22] - **Impact of Legislation**: McKesson does not foresee direct impacts from recent legislative changes regarding drug pricing, as they continue to negotiate fair value for their services [24][25] Future Opportunities - **Clinical Trials and Research**: McKesson sees potential in expanding clinical trial access and management through its Sarah Cannon Research Institute joint venture, which could enhance its oncology platform [33][34] - **New Therapeutic Areas**: The company is exploring opportunities in other therapeutic areas with high innovation and drug spend, beyond oncology and ophthalmology [44][45] Operational Considerations - **Working Capital Management**: McKesson is focused on efficient working capital management, ensuring that fixed fees for services remain unaffected by fluctuations in drug prices [28][29] - **GPO and MSO Dynamics**: The Group Purchasing Organization (GPO) services a wide range of providers, and the company is looking to grow beyond drug distribution into additional service offerings [49] Conclusion - McKesson is strategically positioned to leverage its capabilities in the North American pharmaceutical market, focusing on specialty drugs, operational efficiency through automation, and expanding its oncology services while navigating the evolving regulatory landscape. The company anticipates continued growth and opportunities in high drug spend areas, supported by its investments in technology and clinical services.

Summary of United Therapeutics Conference Call Company Overview - **Company**: United Therapeutics (NasdaqGS: UTHR) - **Event**: UBS Healthcare Conference - **Date**: November 10, 2025 Key Highlights Financial Performance - **Third-Quarter Earnings**: Achieved double-digit year-over-year growth in the Tyvaso franchise [4][9] - **Revenue Target**: Aiming for a $4 billion revenue run rate by the end of 2027, supported by clarity from the TETON-2 trial results [6][7] Product Developments - **Tyvaso Franchise**: Continued growth driven by both nebulizer and dry powder inhaler (DPI) formats, with a focus on PH-ILD [9][10] - **Higher-Dose Cartridges**: Introduction of 80, 96, and 112 microgram cartridges to enhance patient convenience and dosing flexibility [11][13][14] - **TETON-2 Clinical Trial**: Reported as the best IPF study conducted, with promising efficacy and safety data [4][25][26] - **Ralinepag**: Upcoming data readout expected in the first half of 2026, with potential to show improvement over existing therapies [45][50] Market Dynamics - **Competitive Landscape**: Liquidia's launch of Yutrepia has not materially impacted Tyvaso's growth; instead, it is believed to expand the overall market for PAH and PH-ILD [16][20] - **Underdiagnosis of PAH**: The presence of more products is expected to improve diagnosis rates and expand the patient population [18][21] Clinical Trials and Regulatory Updates - **Xenotransplantation Program**: First patient transplanted under IND approval, with a two-cohort study plan; results from the first cohort will dictate the next steps [54][56][72] - **Legal Case Update**: Ongoing litigation regarding PH-ILD; no comments on specifics, but an outcome is expected soon [22][24] Future Outlook - **Pipeline Confidence**: Anticipation of significant trial outcomes in the first half of 2026 for both TETON-1 and ralinepag [41][45] - **Market Potential**: The company believes there is ample opportunity for multiple players in the xenotransplantation market, given the large patient population in need [73] Additional Insights - **Patient-Centric Approach**: Emphasis on patient convenience and the ability to start treatment quickly without extensive pre-requisites [34][36] - **Long-Term Vision**: The company is focused on addressing the significant healthcare costs associated with dialysis and improving patient quality of life through innovative solutions [73] This summary encapsulates the key points discussed during the United Therapeutics conference call, highlighting the company's performance, product developments, market dynamics, and future outlook.

Summary of BeOne Medicines FY Conference Call Company Overview - **Company**: BeOne Medicines (NasdaqGS:BGNE) - **Industry**: Biotechnology - **Key Achievement**: First year of profitability and recognized as one of the fastest-growing large biotech companies [3][4] Competitive Advantages - **Integrated Development**: Fully integrated CRO-free clinical development organization with over 3,600 professionals [4] - **Product Pipeline**: Deep product pipeline with 10 internally developed New Molecular Entities (NMEs) entering the clinic in 2024 and 16 to date [3] - **Cost Efficiency**: 70% of the cost to develop medicines is in clinical development; BeOne aims to reduce this through its integrated approach [4] Product Performance - **Brukinsa**: - Leading market share in the BTK inhibitor market with 47% year-over-year growth in the U.S. and 71% growth in Europe [6][8] - Demonstrated durable progression-free survival (PFS) with 74% landmark PFS at 72 months [6][7] - Significant real-world impact and prescription growth globally [8][9] Market Dynamics - **Fixed Duration Treatments**: - BeOne supports finite treatments that meet four criteria: deep response, sustained PFS, acceptable safety profile, and convenience [10][11] - Current market dynamics show continuous use BTK inhibitors capturing about 50% of the market, with opportunities for growth through fixed-duration offerings [11][12] Pipeline Developments - **Sonrotoclax**: - Designed to be more potent and selective than venetoclax, with a half-life of five hours [14][15] - Breakthrough designation in relapsed refractory MCL, with plans for global filing based on upcoming data [30] - Phase 3 study planned for multiple myeloma, targeting the translocation 11;14 population [32][33] - **BDK CDAC**: - A degrader molecule with a different mechanism, potentially effective against mutations that standard inhibitors cannot target [24][25] - Phase 2 cohort fully enrolled, with data expected in the first half of next year [26] Competitive Landscape - **Comparison with Competitors**: - BeOne's zanubrutinib shows superior response rates compared to pirtobrutinib and acalabrutinib in specific patient populations [20][21] - Ongoing head-to-head studies to validate BeOne's offerings against competitors [16][17] Future Outlook - **Clinical Trials**: - Upcoming data presentations at ASH for various products, including BDK CDAC and sonrotoclax [28][29] - Commitment to advancing solid tumor pipeline, particularly CDK4/6 inhibitors, with a focus on first-line breast cancer [34][36] Conclusion - BeOne Medicines is positioned strongly within the biotechnology sector with a robust product pipeline, innovative clinical development strategies, and a commitment to addressing patient needs through differentiated therapies. The company is actively pursuing growth opportunities in both hematology and solid tumors while maintaining a competitive edge against established players in the market.

Summary of Praxis Precision Medicines FY Conference Call Company Overview - **Company**: Praxis Precision Medicines (NasdaqGS:PRAX) - **Date of Conference**: November 10, 2025 - **Key Executives Present**: CEO Marcio Souza, SVP Finance Lauren, Matt Krogo Key Points Discussed Pipeline and Upcoming Developments - The company is preparing for a potential New Drug Application (NDA) filing in the next few months, particularly for essential tremors (ET) and ulixacaltamide [6][10] - There is a focus on the Essential 3 program, which has shown positive results, and the company is looking to leverage these results for future drug applications [7][8] - The company is moving forward with the SCN2A EMBOLD program, with plans for an NDA submission early next year [9][10] FDA Interactions - The upcoming FDA meeting is crucial for discussing the NDA submission and the adequacy of the evidence from the Essential 3 program [12][15] - The company has had multiple interactions with the FDA, which have been positive, indicating good continuity in the review process [11][20] - The key question for the FDA meeting is whether the current data package constitutes substantial evidence of effectiveness for approval [15][16] Market Opportunity - There are approximately 7 million Americans living with essential tremor, with at least 2 million actively seeking treatment, indicating a significant market potential [37][38] - The company estimates that with conservative market penetration, potential sales could reach $10 billion in the U.S. alone, and $13 billion to $15 billion when including ex-U.S. markets [39][40] - The pricing strategy is expected to be competitive, with a potential 25%-50% discount compared to existing treatments [38][39] Commercial Strategy - The company is building infrastructure to better understand and recruit patients, aiming to expand its database from 200,000 to about 1 million patients before launch [41][42] - The commercial organization is being strengthened, leveraging the experience of executives who have previously launched multiple drugs [42][43] Other Assets and Future Plans - The company is also focusing on relutrigine, which has received breakthrough designation, and is preparing for an interim analysis that could lead to an NDA submission [44][46] - The market for SCN2A and 8A is projected to be significant, with estimates of over $1 billion at launch [50][51] - Upcoming data from the RADIANT study is expected to provide insights into the effectiveness of relutrigine in generalized patients [54][55] Additional Insights - The company emphasizes the importance of FDA's role in ensuring drug safety and efficacy, and it aims to provide substantial evidence to support its applications [20][21] - There is a recognition of the need to overcome biases in market assumptions to accurately reflect the potential of their treatments [37][39] This summary captures the essential discussions and strategic directions of Praxis Precision Medicines as presented in the conference call.

Summary of FireFly Metals Conference Call Company Overview - **Company**: FireFly Metals (OTCPK:MNXM.F) - **Location**: Newfoundland, Canada - **Current Resource**: 59 million tonnes at 2% copper equivalent Key Points and Arguments Resource Development - The company is currently drilling 650 meters in front of its existing resource, yielding 49 meters at 6.1% copper equivalent, which will be included in an upcoming resource update by year-end [1][3] - The strategy focuses on demonstrating the world-class scale and continuity of the deposit, with over 2 km of underground development completed [3][4] - Six drilling rigs are operational, with four for infill drilling and two for extensional drilling, indicating a significant investment in resource expansion [4] Resource Update Expectations - The upcoming resource update is expected to show continued growth in the overall size of the resource and a conversion of inferred resources into measured and indicated categories, aiming for 70% conversion [4][5] - The company anticipates a strong economic potential due to high grades and thicknesses of mineralization, with a focus on early payback and cash flow [5][6] Economic Context - Current market prices for copper are above $11,000 per tonne and gold is at record highs above $4,300 per ounce, enhancing the economic viability of the project [7] - The existing mill has a capacity of 500,000 tonnes per annum, which is insufficient for the large-scale ore body, prompting plans for a new, appropriately scaled mill [8][9] Mining Methodology and Infrastructure - The company plans to adopt bulk mining methods suitable for the ore body, with decline development already reaching 900 meters below the surface [9][10] - Ground conditions are reported to be exceptional, allowing for deeper mining operations, with comparisons to other successful Canadian mines [10] Metallurgical Improvements - Recent metallurgical test results show copper recovery rates of 98% and gold recovery improved from 60% to 85% through a dedicated processing plant, significantly impacting project economics [11] Financing and Exploration Potential - The company is exploring non-dilutive financing options and is moving rapidly towards production [12] - Regional exploration is a key focus, with the potential for discovering additional deposits, as the area has a history of multiple mines [12][13] Upcoming Catalysts - Key upcoming events include the release of drill results, the resource update by year-end, and mining studies in the first half of next year [14] Additional Important Information - The support from local communities and government is described as unprecedented, with environmental approvals obtained in just 45 days [1][2] - The Fraser Institute ranks Newfoundland among the top six jurisdictions globally for government policy, highlighting its favorable operating environment [2]

Kymera Therapeutics FY Conference Summary Company Overview - **Company**: Kymera Therapeutics (NasdaqGM:KYMR) - **Focus**: Development of oral degrader medicines targeting immune inflammatory diseases, particularly through the lead program KT-621, an oral STAT6 degrader Key Points and Arguments Investment Strategy and Pipeline - Kymera aims to build a commercial-stage global company, emphasizing the importance of developing multiple assets rather than relying on a single product [4][5] - The company is committed to advancing a pipeline that addresses difficult questions in immunology, with KT-621 as the top priority, followed by KT-579 and other preclinical programs [6] KT-621 Development Insights - The program targets STAT6 degradation, which is crucial for blocking IL-4 and IL-13 signaling, with preclinical studies showing that degrading STAT6 by 90% or more can match the efficacy of existing biologics like dupilumab [8][9] - Initial human studies demonstrated successful STAT6 degradation and safety, exceeding expectations in terms of biomarker impact compared to dupilumab [10][11] Clinical Study Design - Kymera is conducting a 28-day atopic dermatitis study without a placebo to expedite the development timeline while still gathering meaningful biomarker data [12][15] - The focus is on TH2 biomarkers, particularly TARC, which is expected to show significant changes in patients with atopic dermatitis [16][17] Comparison with Competitors - The only direct competitor identified is dupilumab, as both target IL-4 and IL-13 pathways [24] - Historical data indicates that the baseline severity of conditions in patients has shifted, affecting placebo response rates, but this does not materially impact the efficacy comparison between drugs [22][24] Future Directions and Business Development - Kymera plans to maintain control over KT-621's development through phase 2B studies, with sufficient cash reserves to fund operations into the second half of 2028 [29][30] - The company is open to partnerships if they believe it will maximize the asset's value, but the current focus remains on executing the clinical studies [30] Additional Important Insights - The company emphasizes the importance of understanding the translation of drug effects from preclinical to clinical settings, particularly regarding dose-response relationships [27][28] - Kymera's approach to drug development is characterized by a comprehensive assessment of both pharmacokinetics and biological impact, ensuring that clinical endpoints align with biomarker changes [18][19] This summary encapsulates the critical aspects of Kymera Therapeutics' current strategies, pipeline developments, and market positioning as discussed in the conference.

Summary of Rumble Update Call Company and Industry Overview - **Company**: Rumble - **Industry**: Technology, specifically focusing on video platforms, AI infrastructure, and decentralized financial tools Key Points and Arguments 1. **Transformational Milestones**: Rumble announced three significant milestones: - Acquisition of Northern Data, positioning Rumble as a leader in AI infrastructure with over 22,000 GPUs and nine data centers [3][5] - Tether's $150 million GPU purchase agreement, providing immediate revenue scale and validating Rumble's platform [3][6] - Tether's $100 million advertising commitment to enhance creator monetization on Rumble [3][7] 2. **Market Positioning**: Rumble is redefining itself from a video platform to a comprehensive tech ecosystem that competes with major players like Microsoft, Google, and Amazon [4][8] 3. **User Engagement**: Rumble has 47 million monthly active users and an advertising ecosystem capable of handling over a billion ad requests daily [4][5] 4. **Future Plans**: Rumble aims to expand into web tools and a productivity suite, including email and file storage, to further enhance its ecosystem [5] 5. **Strategic Alignment with Tether**: The partnership with Tether is seen as a strategic alignment that will provide Rumble with the necessary infrastructure and financial backing to grow aggressively [6][11] 6. **Advertising and Revenue Growth**: The $100 million advertising commitment from Tether is expected to significantly boost creator growth and platform monetization [7][36] 7. **Seasonal User Trends**: Rumble experienced a seasonal decline in users during the summer months due to creator inactivity, but anticipates a rebound as creators return to the platform [35] 8. **Creator Acquisition Strategy**: With Tether's advertising support, Rumble plans to aggressively pursue new creators, changing the dynamics of creator acquisition and profitability [36][37] Additional Important Content 1. **Decentralization Vision**: Both Rumble and Tether emphasize the importance of decentralization and freedom in technology, aiming to create an open infrastructure that counters the control of big tech [11][12][13] 2. **Global Financial Inclusion**: Tether's CEO highlighted the role of stablecoins in facilitating remittances and financial access for underserved populations, aligning with Rumble's mission to empower creators and communities [24][30] 3. **Long-term Growth Mandate**: Rumble's focus is on aggressive growth while maintaining a commitment to a free and open internet, contrasting with traditional big tech models [8][15] 4. **Non-Custodial Wallet**: The upcoming Rumble Wallet will be a non-custodial wallet, allowing users to engage in various financial transactions beyond just tipping creators [23][24] 5. **Market Dynamics**: The call addressed the need for a counterbalance to the centralization of information and financial systems, advocating for a technology ecosystem that empowers individuals [30][31] This summary encapsulates the key insights and strategic directions discussed during the Rumble Update Call, highlighting the company's ambitions and the transformative partnerships that are set to redefine its market presence.

MannKind Corporation Investor Call Summary Company Overview - **Company**: MannKind Corporation (NasdaqGM: MNKD) - **Date of Call**: November 10, 2025 - **Focus**: Clinical trial update regarding MannKind's product candidates, specifically the discontinuation of the ICON one Phase three clinical trial for nebulized clofazamine inhalation suspension (MannKind-one hundred one) for treating refractory non-tuberculosis mycobacterium (NTM) lung disease [1][2][4] Key Points and Arguments 1. **Discontinuation of Clinical Trial**: The ICON one Phase three clinical trial was discontinued due to futility, as none of the first 46 participants showed evidence of sputum culture conversion [4][5][6] 2. **Acquisition Background**: MannKind acquired Q Rum Pharma in 2020, which had developed the nebulized formulation of clofazamine, with plans to transition to a dry powder formulation [4] 3. **Safety Profile**: The Data Safety Monitoring Board (DSMB) did not identify any safety concerns during the study, indicating that the lack of efficacy is likely related to the nebulized formulation rather than the molecule itself [6][9] 4. **Next Steps**: MannKind is investigating the reasons for the unexpected trial outcome, focusing on the suspension formulation and its handling instructions [7][8] 5. **Future Development**: The company is advancing the MannKind-one hundred two dry powder formulation of clofazamine towards Phase one trials, with hopes for its efficacy in treating NTM lung disease [8][9] 6. **DPI Candidate Confidence**: There is increased confidence in the dry powder inhaler (DPI) formulation due to better predictability of delivered doses compared to the nebulized formulation, which requires more patient intervention [13][21] 7. **Partial Conversions Observed**: While there were no complete sputum culture conversions, some partial conversions were noted, and the team is analyzing patient-reported outcomes for further insights [14][15][34] 8. **Regulatory Engagement**: MannKind plans to meet with the FDA to discuss findings and next steps for the DPI formulation, with updates expected in Q2 2026 [29][30] Additional Important Information 1. **Human Capital Investment**: The company has invested significant resources in developing both nebulized and DPI formulations, reflecting its commitment to innovative patient-centric solutions [5] 2. **Training and Preparation Issues**: The trial's failure may be linked to improper handling and preparation of the nebulized formulation, highlighting the importance of adherence to protocols [20][22] 3. **Ongoing Research**: MannKind is conducting a 1b trial for another product (02/2001) in idiopathic pulmonary fibrosis (IPF) patients, which is expected to provide insights into the tolerability of the dry powder formulation [35][36] 4. **Market Position**: Despite the setback, MannKind remains committed to developing therapies for serious lung diseases and is monitoring the competitive landscape for potential impacts on its pipeline [40]

Summary of REGENXBIO FY Conference Call Company Overview - **Company**: REGENXBIO (NasdaqGS: RGNX) - **Event**: FY Conference on November 10, 2025 - **Speakers**: CEO Curran Simpson and CMO Steve Pakola Key Points Industry and Product Development - REGENXBIO has been working on AAV (Adeno-Associated Virus) delivery for over 15 years, contributing to significant products like Zolgensma [3][4] - The company is focused on late-stage assets, with a BLA (Biologics License Application) review for the Hunter program targeting MPS II disease, with a PDUFA date set for February [3][4] - The Duchenne program has completed enrollment for its pivotal cohort, with top-line data expected in Q2 next year [4][19] - REGENXBIO is also involved in a partnership with AbbVie for the RGX-314 program targeting wet AMD, with top-line data expected by the end of 2026 [4][7] Clinical Trials and Data Expectations - The RGX-314 program is noted as one of the largest gene therapy clinical trials ever conducted, focusing on non-inferiority against existing anti-VEGF treatments [5][6] - The company aims to demonstrate a decrease in treatment burden and improved vision outcomes through its gene therapy approach [7][16] - The Duchenne program aims to replicate natural dystrophin closely, with a focus on the C-terminal domain for better efficacy [19][20] Commercial Strategy - AbbVie will lead the commercialization of the RGX-314 program, with a 50/50 profit share agreement [11][12] - REGENXBIO does not intend to build its own sales force, relying on AbbVie’s extensive commercial infrastructure [12] Market Needs and Compliance - The primary issue addressed by the RGX-314 program is patient compliance, as many patients struggle with frequent injections [13][16] - The company highlights the significant unmet need for less frequent injections, which has been shown to improve patient outcomes [15][16] Manufacturing and Cost of Goods - REGENXBIO has invested heavily in manufacturing, aiming for cost of goods to align more with biologics than traditional gene therapy [18] - The company reports good yields from its bioreactor process, which is crucial for meeting the demands of the market [18] Regulatory Interactions - The company has maintained consistent interactions with the FDA, with a focus on the Hunter program and the Duchenne protocol [24][29] - A major amendment was triggered for the Hunter program, moving the PDUFA date from November to early February [29] Safety and Efficacy - The company has implemented a targeted immunosuppression regimen to improve safety outcomes, reporting no liver injuries in treated patients [22][23] - The results from the Duchenne program have shown unexpected improvements in older patients, which is a positive indicator for the therapy's efficacy [25][27] Future Expectations - REGENXBIO is optimistic about the potential for traditional approval for the Hunter program, given the robust clinical data provided [32] - The company is also considering pursuing a Priority Review Voucher (PRV) for the Hunter program if approved before September 2026 [35] Conclusion - REGENXBIO is positioned for a significant year ahead with multiple key data readouts and regulatory milestones, focusing on innovative gene therapies that address critical unmet needs in rare diseases [3][4][32]