Core Insights - Q32 Bio Inc. is a clinical stage biotechnology company focused on developing biologic therapeutics aimed at restoring immune homeostasis [3][4] - The company will participate in a fireside chat at Guggenheim's Inaugural Healthcare Innovation Conference on November 12, 2024 [1] Company Overview - Q32 Bio develops biologic therapeutics targeting regulators of the innate and adaptive immune systems to address autoimmune and inflammatory diseases [3] - The lead programs focus on the IL-7 / TSLP receptor pathways and the complement system, which are crucial for re-balancing immunity [3] Product Pipeline - The lead product, bempikibart (ADX-914), is a fully human anti-IL-7Rα antibody being evaluated in two Phase 2 trials for atopic dermatitis and alopecia areata [4] - The second product, ADX-097, targets the complement system with a novel platform that allows tissue-targeted regulation without long-term systemic blockade, differentiating it from current therapies [4] - ADX-097 has completed a Phase 1 ascending dose clinical study in healthy volunteers [4]

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].

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 September 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 Charte ...

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].