• An early-stage **Sarepta Therapeutics**, focused on gene therapies for rare neurological diseases.
• A **CRISPR Therapeutics** or **Intellia Therapeutics** that develops AAV gene therapies for severe rare neurological conditions.

• PBGM01: An investigational gene therapy designed to treat infantile GM1 gangliosidosis.
• PBFT02: A gene therapy candidate aimed at treating frontotemporal dementia (FTD) caused by granulin (GRN) mutations.
• PBMN01: An investigational gene therapy being developed for metachromatic leukodystrophy (MLD).

Passage Bio (PASG) is a clinical-stage genetic medicines company focused on developing therapies for rare neurological diseases. As of their latest financial filings (e.g., their Annual Report on Form 10-K), the company has not yet commercialized any products and explicitly states that it has not generated any revenue from product sales.

Consequently, Passage Bio does not currently have "major customers" in the traditional sense, whether other companies or individuals. Their primary activities are research, development, and conducting clinical trials for their product candidates.

Their funding primarily comes from:

• Investors: Through equity offerings and other financing activities.
• Potential Future Partnerships: While none are currently generating product revenue, like many biotech companies, Passage Bio may pursue collaborations or licensing agreements with larger pharmaceutical companies in the future for co-development or commercialization. If successful, such partners would then represent their "customers" in a business-to-business relationship.

Since Passage Bio is pre-commercial, it does not sell products or services primarily to other companies or individuals at this time.

• Catalent, Inc. (CTLT)

William Chou, M.D. President and Chief Executive Officer

Dr. Chou has served as Chief Executive Officer of Passage Bio since October 2022. He has nearly two decades of healthcare experience across development and commercialization roles. Prior to Passage Bio, he was CEO of Aruvant Sciences, a clinical-stage biopharmaceutical company focused on developing gene therapies for rare diseases, from November 2019 to October 2022. From May 2008 to October 2019, Dr. Chou held various leadership roles at Novartis, including Vice President, Global Disease Lead for its Cell and Gene Therapy unit, where he oversaw the global commercial launch of Kymriah®, the first CAR-T cell therapy. He also led the Kymriah® lymphoma clinical development program. Before Novartis, Dr. Chou worked at the Boston Consulting Group, focusing on commercial and clinical pharmaceutical strategy.

Kathleen Borthwick Chief Financial Officer

Ms. Borthwick was appointed Chief Financial Officer of Passage Bio on March 1, 2024. She previously served as Senior Vice President and Interim Chief Financial Officer from July 2023 to February 2024, and as Vice President of Finance from November 2021 to July 2023. Before joining Passage Bio, Ms. Borthwick held various leadership roles in Finance at Johnson & Johnson from June 1997 to October 2021, supporting Research & Development, Manufacturing, Business Development, Treasury, and commercial operations for global Pharmaceutical and Medical Technology business segments.

Sue Browne, PhD Chief Scientific Officer

Eden Fucci SVP Technical Operations

Stuart Henderson Chief Business Officer

Key Risks to Passage Bio (PASG):

1. Clinical Trial and Regulatory Approval Risks: As a clinical-stage genetic medicines company, Passage Bio's success is highly dependent on its ability to successfully develop, conduct clinical trials, and obtain regulatory approval for its product candidates. There is a significant risk that preclinical studies and clinical trials may not yield positive results or may experience delays. Regulatory authorities may also require additional information, further studies, or could delay or fail to approve the company's drug candidates. Furthermore, positive results in early-stage trials may not be predictive of success in later-stage or pivotal trials.
2. Operating Losses and Need for Additional Funding: Passage Bio has a history of operating losses and is currently unprofitable, anticipating continued losses for the foreseeable future. The company is quickly burning through cash and suffers from weak gross profit margins. While the company currently projects its cash, cash equivalents, and marketable securities to fund operations into the first quarter of 2027, it will ultimately need to secure additional funding to continue its research, development, and commercialization efforts.
3. Intellectual Property and Third-Party Dependence: Passage Bio faces risks related to its ability to protect and enforce its intellectual property and other proprietary rights. Additionally, the company is dependent on collaborators and other third parties for various aspects of its business, including the development and manufacture of product candidates, which are outside of its direct control.

Passage Bio's main product pipeline includes therapies for several rare central nervous system disorders. The addressable market sizes for some of these products are as follows:

• PBGM01 for GM1 Gangliosidosis: The global GM1 Gangliosidosis Treatment market is estimated to be valued at USD 142.1 million in 2025 and is projected to reach USD 651.7 million by 2032.
• PBKR03 for Krabbe Disease: The market for Krabbe disease in the 7 major markets (US, Germany, France, Italy, Spain, UK, and Japan) was valued at USD 2.6 billion in 2024 and is expected to reach USD 5.1 billion by 2035.
• PBML04 for Metachromatic Leukodystrophy (MLD): The global Metachromatic Leukodystrophy Treatment market size was valued at USD 2.3 billion in 2024 and is projected to reach USD 4.11 billion by 2035.

Expected Drivers of Future Revenue Growth for Passage Bio (PASG)

Share Issuance

• Passage Bio completed its Initial Public Offering (IPO) on February 28, 2020, raising $126 million by issuing 7,400,000 shares at a price between $16.00 and $18.00 per share.
• A 1-for-20 reverse stock split was implemented, becoming effective on July 14, 2025, which reduced the number of outstanding common shares from approximately 62.4 million to about 3.12 million.
• The company has a remaining capacity of $15.8 million under its At-The-Market (ATM) facility, indicating potential for future equity offerings.

Inbound Investments

• Passage Bio has a strategic collaboration and licensing agreement with the University of Pennsylvania's Gene Therapy Program for discovery and IND-enabling preclinical work.
• The company received $9.7 million in non-refundable sublicense and transition services payments related to amended Gemma sublicenses.

Capital Expenditures

• Capital expenditures amounted to $0.03 million in 2023, $0.15 million in 2022, and $5.27 million in 2021.
• Passage Bio ceased laboratory operations in Hopewell, which resulted in a $2.6 million impairment of long-lived assets and the subsequent sale of substantially all lab equipment for $1.2 million.