UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___________________ to ___________________ Commission File Number: 001-38433 Q32 Bio Inc. (Exact name of registrant as specified in its charter) Delaware 47-3468 ...

Exhibit 99.1 Q32 Bio Reports Second Quarter 2024 Financial Results and Provides Corporate Update -- Completed enrollment in bempikibart atopic dermatitis (AD) Phase 2 clinical trial and increased trial size to 121 patients based on Part B patient demand -- -- Bempikibart Phase 2 topline results in AD and alopecia areata (AA) remain on-track for Q4'24 -- -- Enrolling patients in the Phase 2 basket trial of ADX-097 for complement mediated renal diseases; initial open-label data expected by year-end 2024 and t ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___________________ to ___________________ Commission File Number: 001-38433 Q32 Bio Inc. (Exact name of registrant as specified in its charter) Delaware 47-346 ...

Exhibit 99.1 Q32 Bio Reports First Quarter 2024 Financial Results and Provides Corporate Update -- Completed enrollment in bempikibart alopecia areata (AA) Phase 2 clinical trial, with topline results expected in Q4'24; enrollment in bempikibart atopic dermatitis (AD) Phase 2 clinical trial remains ontrack, with topline results expected in Q4'24 -- -- ADX-097 continues to advance, with anticipated Phase 2 clinical trial initiations on-track: renal basket in 1H'24 and ANCA-associated vasculitis (AAV) in 1H'2 ...

Financial Performance - The company has incurred significant losses since inception and anticipates continued losses for the foreseeable future, which may affect the market value of its common stock[8]. - The company raised approximately $721 million in net proceeds since inception, including a $130 million upfront cash payment from the agreement with Oxford Biomedica[23]. - The company reduced its workforce by 86% to significantly lower ongoing operating costs while evaluating strategic alternatives[10]. Strategic Partnerships and Mergers - An Agreement and Plan of Merger was entered into with Q32 Bio Inc., with Q32 having an aggregate equity value of $195 million and the company's equity value expected to be approximately $80 million[11][12]. - Q32 is expected to complete a Concurrent Financing with gross proceeds of $42 million prior to the Merger[13]. - OXB (US) LLC was established in 2022 to support scalable AAV manufacturing, achieving high-quality titers of E15 vg/L and over 90% fully intact vector[77]. Clinical Trials and Product Development - The company has withdrawn all clinical trial applications for its investigational gene therapy candidates, including HMI-102 and HMI-203, and terminated all related clinical studies[17]. - Clinical data from the pheEDIT trial indicated that HMI-103 was generally well-tolerated, with participant 1 achieving a reduction in plasma phenylalanine levels below the treatment guideline threshold[17]. - The company is exploring strategic alternatives for its product candidates, including potential sales of HMI-103 and HMI-204[17]. - The pheEDIT Phase 1 clinical trial for HMI-103 was inactivated in September 2023, with clinical data showing that HMI-103 was well-tolerated in all three participants, with no serious adverse events reported[59]. - The company has completed IND-enabling studies for HMI-202, a gene therapy for MLD, and announced HMI-204, an optimized one-time gene therapy candidate that showed significant expression of human ARSA in multiple brain regions[18]. Regulatory Environment - The regulatory approval processes for the company's product candidates are lengthy and inherently unpredictable, which may impact future commercialization efforts[8]. - The company must comply with Good Manufacturing Practices (GMP) and conduct stability studies to ensure product quality over its shelf life[94]. - The FDA review process for biologics includes submission of a Biologics License Application (BLA) that must demonstrate safety, purity, and potency[95]. - The company is required to navigate extensive regulatory processes for product approval, which demand significant time and financial resources[90]. Gene Therapy and Gene Editing Technologies - The company focuses on gene therapy and gene editing approaches to address monogenic diseases, aiming for functional gene integration through HR[24]. - The proprietary AAVHSC platform enables nuclease-free gene editing and gene therapy with improved safety profiles and therapeutic efficiencies[29]. - AAVHSCs demonstrate high gene integration efficiencies, achieving therapeutic ranges significantly higher than traditional nuclease-based methods[29]. - The company’s gene editing approach aims to achieve functional gene integration into the patient's genome, potentially addressing the majority of monogenic diseases[24]. Market and Competitive Landscape - The company has paused development of its product candidates, facing competition from major pharmaceutical and biotechnology firms[80]. - Coverage and reimbursement decisions by third-party payors are increasingly restrictive, impacting sales of medical products[108]. - Legislative proposals in the U.S. aim to contain healthcare costs, which may affect the pricing and profitability of pharmaceutical products[111]. Intellectual Property - The company relies on a combination of patents and trade secrets to protect its intellectual property, with a focus on maintaining a competitive advantage[82]. - The patent term for most applications is 20 years, with potential extensions available under the Hatch-Waxman Act[83]. - The company holds 13 granted patents in the U.S. related to novel AAV capsids, expected to expire in 2031, with possible extensions of up to five years[85]. Employee Relations - The company emphasizes the importance of maintaining good relationships with employees, as none are represented by a labor union[136]. - As of December 31, 2023, the company had 7 full-time employees, including one with an M.D. or Ph.D. degree[136].

PART I—FINANCIAL INFORMATION [Item 1. Financial Statements](index=5&type=section&id=Item%201.%20Financial%20Statements.) The company's financial statements reflect a strategic halt in operations, resulting in significant net losses and going concern doubts | Indicator | As of Sep 30, 2023 (in thousands) | As of Dec 31, 2022 (in thousands) | | :--- | :--- | :--- | | Cash and cash equivalents | $29,111 | $33,986 | | Short-term investments | $74,187 | $141,040 | | **Total current assets** | **$106,635** | **$181,015** | | **Total assets** | **$140,063** | **$228,470** | | Total liabilities | $51,852 | $50,492 | | **Total stockholders' equity** | **$88,211** | **$177,978** | | Accumulated deficit | ($525,979) | ($429,137) | | Indicator (in thousands) | Three Months Ended Sep 30, 2023 | Three Months Ended Sep 30, 2022 | Nine Months Ended Sep 30, 2023 | Nine Months Ended Sep 30, 2022 | | :--- | :--- | :--- | :--- | :--- | | Collaboration revenue | $0 | $802 | $1,156 | $2,406 | | Research and development | $17,519 | $25,854 | $60,489 | $71,202 | | General and administrative | $6,842 | $7,810 | $23,355 | $29,991 | | Restructuring and other charges | $6,640 | $0 | $6,640 | $0 | | **Loss from operations** | **($31,001)** | **($32,862)** | **($89,328)** | **($98,787)** | | Gain on sale of business | $0 | $0 | $0 | $131,249 | | **Net income (loss)** | **($32,954)** | **($33,726)** | **($96,842)** | **$29,290** | | **Net income (loss) per share-diluted** | **($0.57)** | **($0.59)** | **($1.68)** | **$0.51** | - In July 2023, the company initiated a review of strategic options, stopped further development of its programs, and reduced its workforce by **86%** to significantly cut operating costs[23](index=23&type=chunk) - Management has concluded there is **substantial doubt** regarding the company's ability to continue as a going concern for more than twelve months from the financial statement issuance date[30](index=30&type=chunk) - A restructuring charge of **$6.9 million** for severance and related costs was recorded in Q3 2023 in connection with the 86% workforce reduction[84](index=84&type=chunk) - Effective October 1, 2023, the company was released from its primary lease obligations, which is expected to result in a gain of **$8.8 million**[132](index=132&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=30&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses its strategic pivot, program halts, and workforce reduction, which significantly lowered operating expenses - In July 2023, the Board approved a plan to explore strategic options, including mergers or asset sales, and consequently stopped all program development and reduced its workforce by **86%**[137](index=137&type=chunk) | Expense Category (in thousands) | Three Months Ended Sep 30, 2023 | Three Months Ended Sep 30, 2022 | Change | | :--- | :--- | :--- | :--- | | Research and development | $17,519 | $25,854 | ($8,335) | | General and administrative | $6,842 | $7,810 | ($968) | | Restructuring and other charges | $6,640 | $0 | $6,640 | - The **$8.3 million decrease** in Q3 2023 R&D expenses compared to Q3 2022 was primarily due to the decision to stop program development and the workforce reduction[167](index=167&type=chunk) - As of September 30, 2023, the company had **$103.3 million** in cash, cash equivalents, and short-term investments, but management has concluded there is **substantial doubt** about its ability to continue as a going concern[151](index=151&type=chunk)[199](index=199&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=42&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's primary market risk is interest rate sensitivity on its cash and investments, with inflation deemed immaterial - The company's main market risk is interest rate sensitivity on its **$103.3 million** of cash and investments; a hypothetical 10% change in interest rates is not expected to have a material effect[206](index=206&type=chunk) - As of September 30, 2023, the company does not believe inflation has had a material effect on its business, financial condition, or results of operations[207](index=207&type=chunk) [Item 4. Controls and Procedures](index=43&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective, with no material changes to internal controls - Based on an evaluation as of the end of the period, the CEO and CFO concluded that the company's disclosure controls and procedures were **effective**[210](index=210&type=chunk) - No changes in internal control over financial reporting occurred during the three months ended September 30, 2023, that have materially affected, or are reasonably likely to materially affect, internal controls[211](index=211&type=chunk) PART II. OTHER INFORMATION [Item 1. Legal Proceedings](index=44&type=section&id=Item%201.%20Legal%20Proceedings) The company is defending a securities class action lawsuit related to its HMI-102 clinical trial disclosures - The company is defending a putative class action lawsuit filed in March 2022 related to disclosures for its HMI-102 clinical trial; a motion to dismiss the case is currently pending[213](index=213&type=chunk) [Item 1A. Risk Factors](index=44&type=section&id=Item%201A.%20Risk%20Factors) Key risks include a history of losses, going concern doubts, strategic uncertainty, and consequences of workforce reduction - The company has a history of significant operating losses, with an accumulated deficit of approximately **$526.0 million** as of September 30, 2023, and anticipates continued losses[216](index=216&type=chunk) - Management has concluded there is **substantial doubt** regarding the company's ability to continue as a going concern for more than twelve months[231](index=231&type=chunk) - The ongoing evaluation of strategic alternatives is costly and complex, with **no assurance** that any transaction will be completed or have a positive impact[218](index=218&type=chunk)[222](index=222&type=chunk) - The recent **86% reduction in force** may lead to unintended consequences such as loss of institutional knowledge and decreased morale[481](index=481&type=chunk)[483](index=483&type=chunk) - The company's novel gene editing and AAVHSC platforms are unproven in late-stage trials, and the regulatory landscape for such therapies is **uncertain and continues to change**[250](index=250&type=chunk)[251](index=251&type=chunk)[253](index=253&type=chunk) [Item 2. Unregistered Sales of Equity Securities, Use of Proceeds, and Issuer Purchases of Equity Securities](index=93&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities,%20Use%20of%20Proceeds,%20and%20Issuer%20Purchases%20of%20Equity%20Securities) This section is not applicable for the reporting period [Item 5. Other Information](index=93&type=section&id=Item%205.%20Other%20Information) The company reports no other material information and notes its status as a smaller reporting company - The company is a **smaller reporting company** and is not required to provide information otherwise required under Item 408(a) of Regulation S-K[527](index=527&type=chunk) [Item 6. Exhibits](index=95&type=section&id=Item%206.%20Exhibits) This section lists all exhibits filed with the Form 10-Q, including corporate governance documents and certifications

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___________________ to ___________________ Commission File Number: 001-38433 Homology Medicines, Inc. (Exact Name of Registrant as Specified in its Charter) De ...

Financial Performance - Net loss for Q1 2023 was $28,844,000, compared to a net income of $92,105,000 in Q1 2022, reflecting a significant change in financial performance[13]. - Loss from operations for Q1 2023 was $27,511,000, compared to a loss of $37,618,000 in Q1 2022, indicating an improvement of about 27%[13]. - For the three months ended March 31, 2023, the company reported a net loss of $28.8 million compared to a net income of $92.1 million for the same period in 2022[20]. - The company incurred a loss from operations of $28.8 million during the three months ended March 31, 2023, and has an accumulated deficit of $458.0 million as of the same date[26]. - The accumulated deficit increased to $457,981,000 as of March 31, 2023, from $429,137,000 at the end of 2022[11]. Revenue and Expenses - Collaboration revenue for the three months ended March 31, 2023, was $802,000, consistent with the same period in 2022[13]. - Total operating expenses decreased to $28,313,000 in Q1 2023 from $38,420,000 in Q1 2022, representing a reduction of approximately 26%[13]. - Research and development expenses for Q1 2023 were $19,988,000, down from $24,273,000 in Q1 2022, a decrease of approximately 18%[13]. - General and administrative expenses also decreased to $8,325,000 in Q1 2023 from $14,147,000 in Q1 2022, a reduction of about 41%[13]. - The company did not generate any revenue from product sales and does not expect to do so in the foreseeable future[26]. Cash Flow and Liquidity - Cash and cash equivalents decreased to $39.5 million as of March 31, 2023, down from $248.4 million at the end of the same period in 2022[36]. - The company recorded a net cash used in operating activities of $26.1 million for the three months ended March 31, 2023, compared to $30.7 million for the same period in 2022[20]. - The company provided $31.4 million in net cash from investing activities during the three months ended March 31, 2023, compared to $168.3 million in the same period in 2022[20]. - As of March 31, 2023, the company believes its cash and cash equivalents will enable it to continue operations for at least one year[27]. - The company expects to incur additional operating losses and negative operating cash flows for the foreseeable future[26]. Stockholders' Equity and Investments - Total stockholders' equity decreased to $151,899,000 as of March 31, 2023, down from $177,978,000 at the end of 2022[11]. - The Company’s total short-term investments amounted to $110.571 million, with $31.297 million in commercial paper, $57.202 million in US Treasury securities, and $22.072 million in corporate debt securities[63]. - The company has filed a Registration Statement for the registration of up to $250.0 million of its common stock, with $75.0 million available for sale under an "at-the-market" offering[24]. - The company recorded a gain of $131.2 million on the sale of its manufacturing business, with cash received amounting to $130.0 million and a fair value of equity method investment at $31.2 million[80]. Lease and Operating Costs - Operating lease costs for the three months ended March 31, 2023, were $1.1 million, compared to $0.9 million for the same period in 2022[88]. - The company has a noncancelable operating lease for approximately 67,000 square feet, with an initial annual base rent of $39.50 per square foot, increasing by three percent annually[83]. - Total undiscounted lease payments amount to $43.646 million, with a present value of operating lease liabilities at $29.101 million[89]. - Cash paid for lease liabilities in Q1 2023 was $1.101 million, an increase from $0.877 million in Q1 2022[91]. Risks and Future Outlook - The company anticipates continued losses for the foreseeable future and will require additional capital to fund operations[6]. - The company is heavily dependent on the success of its product candidates, with no history of commercializing genetic medicine products, which poses significant risks[6]. - The company does not believe inflation has materially affected its financial condition as of March 31, 2023, but acknowledges potential future impacts on expenses[204]. Stock-Based Compensation - The Company recorded stock-based compensation expense of $2.369 million in Q1 2023, down from $4.051 million in Q1 2022[106]. - The Company expects to recognize $15.8 million of unrecognized compensation expense related to unvested share-based compensation over approximately 2.7 years[106]. - The fair value of stock options granted in Q1 2023 was estimated at $1.06 per share, compared to $1.78 in Q1 2022[101]. - The total intrinsic value of options exercised during Q1 2022 was insignificant, with no options exercised in Q1 2023[101]. Collaboration and Agreements - Under the Supply Agreement with OXB Solutions, the company is committed to purchase approximately $29.7 million worth of AAV-based products in 2023[117]. - The company recorded purchases from OXB Solutions of $4.9 million for drug substance and $1.2 million for process development services during the three months ended March 31, 2023[118]. - The exclusive right of first refusal (ROFR) granted to Pfizer for collaboration on HMI-102 and HMI-103 expired on May 9, 2023[111].

Clinical Trials and Product Development - The company has initiated a Phase 1 clinical trial for HMI-103, its lead gene editing candidate for classical PKU, with the first participant dosed in January 2023 and additional patients being screened across ten active sites[10]. - The HMI-103 trial is expected to enroll up to nine patients aged 18-55, with initial data anticipated by mid-2023, focusing on safety and serum phenylalanine changes[11]. - HMI-203, an investigational gene therapy for Hunter syndrome, is currently in a Phase 1 trial with initial data expected in the second half of 2023, targeting nine male patients aged 18-45[13]. - The company paused enrollment in the HMI-102 trial to prioritize the HMI-103 trial, despite observing biologic activity in the former[14]. - HMI-204 is an optimized gene therapy candidate for MLD, with IND-enabling studies completed and the company seeking a partner for advancement[86]. - The company has initiated a Phase 1 trial with HMI-103 for the treatment of classical PKU, with additional patients being screened across ten active clinical trial sites[36]. - The company plans to enroll younger patients in subsequent HMI-103 clinical trials if positive safety and efficacy results are established in adults[11]. - HMI-203 showed robust biodistribution and significant reductions in heparan sulfate GAG levels in multiple organs in preclinical studies[13]. Financial Overview and Capital Requirements - The company anticipates continued losses for the foreseeable future and will require additional capital to fund operations, which may impact its ability to commercialize product candidates[8]. - The company has raised approximately $721 million in aggregate net proceeds since its inception, including $130.0 million from the Oxford agreement[22]. - The company has incurred significant losses since inception and expects to continue incurring losses for the foreseeable future[8]. - The company requires additional capital to fund operations and may not be able to complete the development and commercialization of its product candidates if financing is not obtained[8]. Regulatory and Compliance Challenges - The regulatory approval processes for its product candidates are lengthy and unpredictable, posing a risk to the company's business[8]. - The FDA approval process for biological products requires extensive regulatory compliance, including preclinical and clinical trials, and submission of a Biologics License Application (BLA)[119]. - The company must navigate various regulatory requirements and may incur substantial time and financial resources in obtaining approvals[119]. - The FDA reviews Biologics License Applications (BLAs) to ensure products are safe, pure, potent, and effective, typically adding approximately two months to the review timeline[129]. - Orphan drug designation can be granted for drugs treating rare diseases affecting fewer than 200,000 individuals in the U.S., providing financial incentives such as grant funding and tax advantages[130]. Intellectual Property and Competitive Landscape - The company has a robust intellectual property portfolio with issued patents for its family of 15 AAVHSCs, which is considered a strategic asset[22]. - The company has exclusive worldwide rights to its technologies, including 15 issued composition of matter patents in the United States for its novel AAVHSCs[37]. - The company has entered into a license agreement with Caltech, paying an initial licensing fee of $100,000 and up to $7.2 million in milestone payments for the first licensed product[115]. - The company relies on a combination of patents, trade secrets, and confidentiality agreements to protect its intellectual property[106]. - The company faces competition from major pharmaceutical and biotechnology companies, academic institutions, and research institutions, with potential competitors including American Gene Technologies and BioMarin[103]. Manufacturing and Supply Chain - The company relies on third-party manufacturers, which increases the risk of supply issues that could delay development or commercialization efforts[8]. - The company closed a transaction with Oxford Biomedica Solutions, resulting in the transfer of assets primarily used for AAV vector manufacturing in exchange for 175,000 common equity units[18]. - The manufacturing strategy utilizes HEK293 cells, ensuring scalability and regulatory familiarity[99]. - The manufacturing capabilities established with Oxford are designed to support the pipeline programs while reducing costs and maintaining quality[37]. Gene Therapy and Technology Platforms - The company focuses on monogenic diseases, utilizing gene therapy and gene editing approaches to address these conditions[23]. - The proprietary AAVHSC platform enables nuclease-free gene editing, which is believed to provide improved safety and efficacy compared to traditional methods[31]. - The AAVHSC platform allows for high-efficiency gene editing without the use of nucleases, simplifying the manufacturing and delivery of therapeutic candidates[33]. - The AAVHSCs demonstrate gene integration efficiencies believed to be in therapeutic ranges, significantly higher than both nuclease-based and other AAV-based approaches[31]. - The AAVHSC technology allows for targeting multiple tissues, including the liver, CNS, and muscle, with a single intravenous administration[49]. Market and Treatment Landscape - The incidence of PKU in the U.S. is estimated at one in 12,707, translating to approximately 350 new cases annually[60]. - Current treatments for PKU, such as Kuvan and Palynziq, do not address the underlying genetic defect, with Kuvan sales at approximately $228 million and Palynziq at $255 million in 2022[64][67]. - The gene integration approach aims to restore PAH activity to 10% or more of normal levels, potentially normalizing serum Phe levels and improving patient quality of life[68]. - Approximately 80% of individuals lack pre-existing neutralizing antibodies against AAVHSCs, enhancing the potential patient pool for gene therapies[57]. - The company is focused on developing product candidates for monogenic diseases with significant unmet medical needs, particularly in the liver, CNS, and peripheral tissues[34].

Financial Performance - Total current assets increased to $213,204,000 as of September 30, 2022, compared to $191,909,000 as of December 31, 2021, representing a growth of 11.5%[17] - Collaboration revenue for the three months ended September 30, 2022, was $802,000, a decrease of 52.2% from $1,677,000 in the same period of 2021[19] - The net loss for the three months ended September 30, 2022, was $33,726,000, compared to a net loss of $30,608,000 for the same period in 2021, reflecting a 10.3% increase in losses[19] - The company reported a comprehensive loss of $34,142,000 for the three months ended September 30, 2022, compared to a comprehensive loss of $30,615,000 for the same period in 2021[23] - For the nine months ended September 30, 2022, the company recorded a net income of $29.3 million, primarily due to a gain from the transaction with Oxford Biomedica[38] - The company reported a net cash used in operating activities of $86.5 million for the nine months ended September 30, 2022, compared to $79.3 million for the same period in 2021[30] - The company expects to incur additional operating losses and negative operating cash flows for the foreseeable future[38] - The company has not generated any revenue from product sales and does not expect to do so in the near future[38] Assets and Liabilities - Cash and cash equivalents decreased to $43,162,000 as of September 30, 2022, from $108,382,000 as of December 31, 2021, indicating a decline of 60.2%[17] - Total liabilities increased to $53,590,000 as of September 30, 2022, compared to $42,070,000 as of December 31, 2021, marking a rise of 27.4%[17] - Stockholders' equity rose to $209,061,000 as of September 30, 2022, from $169,651,000 as of December 31, 2021, an increase of 23.2%[17] - As of September 30, 2022, the company had an accumulated deficit of $394.8 million[38] - The company’s total financial assets amounted to $201.074 million as of September 30, 2022, including cash equivalents of $43.162 million and short-term investments of $157.912 million[76] Revenue and Expenses - Total operating expenses for the nine months ended September 30, 2022, were $101,193,000, up 5.8% from $95,493,000 in the same period of 2021[19] - The company’s stock-based compensation expense for the nine months ended September 30, 2022, was $9.965 million, compared to $12.582 million in 2021[30] - The Company reported operating lease costs of $2.826 million for the nine months ended September 30, 2022, compared to $1.869 million for the same period in 2021, reflecting a year-over-year increase of approximately 51.1%[94] - Research and development expenses for the three months ended September 30, 2022, increased by $1.87 million to $25.85 million compared to $23.99 million in 2021[191] - General and administrative expenses decreased by $541,000 to $7.81 million for the three months ended September 30, 2022[191] Investments and Financing - The company completed a transaction with Oxford Biomedica, receiving $130 million in upfront cash and retaining a 20% ownership interest in the new company[35] - The Company recorded a gain of $131.249 million on the sale of its manufacturing business, with cash received amounting to $130 million and a fair value of equity method investment at $31.223 million[86] - The company has raised approximately $721 million since its inception in 2015, including $130.0 million from the Oxford transaction[158] - The company has $148.4 million of common stock available for sale under its at-the-market offerings as of September 30, 2022[37] - The company entered into a sales agreement in March 2020 to issue and sell common stock with an aggregate value of up to $150 million under the ATM program[215] Clinical Development - The Company is developing multiple clinical-stage product candidates, including HMI-103 for PKU, HMI-203 for MPS II, and HMI-104 for PNH, utilizing its proprietary AAVHSC platform[131] - HMI-103 is currently in a Phase 1 clinical trial, with enrollment expected to include up to nine patients aged 18-55 diagnosed with classical PKU[135] - HMI-203 has received orphan drug designation from both the EMA and FDA, with a Phase 1 trial expected to enroll up to nine male patients aged 18-45 diagnosed with Hunter syndrome[138] - The Company paused enrollment in the HMI-102 trial to focus resources on the HMI-103 trial, despite observing biologic activity in the HMI-102 trial[141] - The Company is actively seeking a partner to advance the preclinical development of HMI-204, an optimized gene therapy candidate for MLD[146] Legal and Compliance - The Company believes the claims in a pending securities class action lawsuit lack merit and has filed motions to transfer venue and dismiss the case[95]