Table of Contents As filed with the Securities and Exchange Commission on April 19, 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 Q32 BIO INC. (Exact name of registrant as specified in its charter) (State or other jurisdiction of incorporation or organization) (Primary Standard Industrial Classification Code No.) Delaware 2834 47-3468154 (I.R.S. Employer Identification Number) 830 Winter Str ...

Since its inception, the Company's operations have been focused on organizing and staffing, business planning, raising capital, establishing the Company's intellectual property portfolio and performing research and development of its product candidates, programs and platform. The Company has primarily funded its operations with proceeds from the sale of convertible preferred stock, convertible notes, venture debt and its collaboration arrangement. The Company is subject to risks and uncertainties common to ...

BEDFORD, Mass., March 18, 2024 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (NASDAQ:FIXX) today announced that it declared a distribution to its common stockholders of record as of the close of business on March 21, 2024 of the right to receive one contingent value right (CVR) for each outstanding share of Homology common stock held by such stockholder as of such record date. The payment date for such distribution is expected to be March 27, 2024 (three business days after the expected closing of the merger ...

FORM 10-K Certain portions of the information required to be furnished pursuant to Part III of this Annual Report on Form 10-K will be set forth in, and incorporated by reference from, the registrant's definitive proxy statement for the annual meeting of stockholders or an amendment to this Annual Report on Form 10-K which will be filed with the Securities and Exchange Commission no later than 120 days after the end of the fiscal year ended December 31, 2023. Auditor Firm Id: 34 Auditor Name: Deloitte & Tou ...

Research and Development Expenses Research and development expenses consist primarily of costs incurred for research activities, including discovery efforts, and the development of our product candidates, and include: • salaries, benefits and other related costs, including stock-based compensation expense, for personnel engaged in research and development functions; • expenses incurred under agreements with third parties, including contract research organizations, or CROs, and other third parties that condu ...

86 The market price of our common stock may be volatile and fluctuate substantially, which could result in substantial losses for purchasers of our common stock. As a public company, we have incurred and expect to continue to incur significant legal, accounting and other expenses that we did not incur as a private company. The Sarbanes-Oxley Act of 2002, the Dodd-Frank Wall Street Reform and Consumer Protection Act, the listing requirements of The Nasdaq Global Select Market and other applicable securities ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___________________ to ___________________ Homology Medicines, Inc. (Exact Name of Registrant as Specified in its Charter) Securities registered pursuant to Section 12(b) of the Act: Title of each class Trading Symbol(s) Name of each exchange on which registered Common Stock, $0.0001 par value per share FIX ...

Clinical Trials and Development - The company paused enrollment in the pheNIX clinical trial for HMI-102 to focus on the Phase 1 pheEDIT clinical trial for HMI-103, which is currently recruiting adult PKU patients[10] - HMI-103 is expected to enroll up to nine patients in three dose cohorts, ages 18-55, with a focus on serum phenylalanine changes and safety endpoints[22] - The company has initiated a Phase 1 trial with HMI-103 for classical PKU, with initial data expected mid-2023, and has dosed the first patient in the trial[47] - A Phase 1 trial for HMI-203, targeting Hunter syndrome, is ongoing with initial data expected in the second half of 2023, involving up to nine male patients[49] - The Phase 1 trial for HMI-203 is expected to enroll up to nine male patients aged 18-45 years with Hunter syndrome, with initial data anticipated in the second half of 2023[127] - The Phase 1/2 pheNIX clinical trial for HMI-102 enrolled six patients across three dose cohorts, with no treatment-related serious adverse events reported[123] - The company paused enrollment in the Phase 1/2 pheNIX trial to focus on the Phase 1 pheEDIT trial, which aims to evaluate in vivo gene editing candidate HMI-103 for PKU[152] - The Phase 2 expansion phase of the pheNIX trial has shown generally well-tolerated results with clinically meaningful reductions in Phe levels and increases in Tyr as of September 30, 2021[152] Regulatory Designations and Approvals - The company received Fast Track Designation from the FDA for HMI-103 for treating neurocognitive and neuropsychiatric manifestations of PKU[23] - HMI-103, the lead gene editing program, is designed to replace the disease-causing gene with a functional gene and has received orphan designations from both the EC and FDA[84] - The FDA lifted the clinical hold on the pheNIX trial on June 13, 2022, after the company satisfactorily addressed all clinical hold issues[152] - Orphan drug designation can be granted for drugs treating rare diseases, providing financial incentives and exclusivity for seven years upon approval[183] - The FDA's Fast Track program expedites the review of biological products intended for serious conditions, with a determination made within 60 days of the sponsor's request[186] - Biological product candidates may also qualify for Breakthrough Therapy designation, which includes more intensive FDA interaction and guidance starting as early as Phase 1[186] - Accelerated approval may be granted for biological products that show meaningful therapeutic benefits over existing treatments, with the requirement of confirmatory clinical studies post-approval[187] - The Regenerative Medicine Advanced Therapy (RMAT) designation allows for more frequent meetings with the FDA and eligibility for rolling review and priority review[187] Financials and Market Value - The aggregate market value of the company's voting and non-voting stock held by non-affiliates was approximately $92.3 million as of June 30, 2022[14] - The company raised approximately $721 million in aggregate net proceeds since its inception in 2015, including a $130 million upfront cash payment from an agreement with Oxford and a $60 million equity investment from Pfizer[55] - The company closed a transaction with Oxford Biomedica Solutions, receiving $130 million for equity units and maintaining a 20% ownership stake in OXB Solutions[52] Gene Therapy and Technology - The company aims to utilize its proprietary AAVHSCs to deliver genetic medicines efficiently and precisely through a single administration[21] - The company is focused on gene integration via homologous recombination to avoid unwanted modifications to the genome[30] - The company is utilizing AAVHSCs to deliver therapeutic DNA for antibody proteins, enabling the formation of fully functional IgG antibodies in the liver[33] - AAVHSCs demonstrate unique biodistribution properties, targeting multiple tissues including the liver, CNS, and muscle, which allows for addressing a broad range of monogenic diseases[35] - The proprietary AAVHSC platform enables gene therapy and gene editing with high precision and efficiency, allowing for the introduction of entire genes or precise repairs of individual mutated nucleotides[62] - The company’s AAVHSC technology allows for gene integration at efficiencies greater than both nuclease-based and other AAV-based approaches, simplifying manufacturing and improving safety[80] - AAVHSCs have shown a statistically significant increase in genome editing efficiencies, with up to 2.8% of alleles edited in treated mice[44] - The genetic medicines platform is designed to perform gene editing at high efficiency without the use of nucleases, simplifying manufacturing and delivery[35] Intellectual Property and Licensing - The company has exclusive worldwide rights to its technologies, including issued composition of matter patents in the United States for 15 novel AAVHSCs[67] - The company has co-exclusively licensed patents for novel AAV capsids and vectors, expected to expire between 2034 and 2036, enhancing its intellectual property portfolio[145] - The company entered a licensing agreement with Caltech, paying an initial fee of $100,000 and up to $7.2 million in milestone payments for the first licensed product, along with low single-digit royalties on net sales[203] - The company is focused on sublicensing the AAV-related patents owned by Caltech, with no current therapeutic product candidates utilizing Caltech technology[203] Manufacturing and Production - The company established OXB Solutions with Oxford to enhance its AAV vector manufacturing capabilities, which is crucial for supporting its product candidates[67] - OXB Solutions' platform produced high-quality titers of 15 E15 vg/L and achieved over 90% fully intact vector, with a manufacturing scale of 2000L in non-GMP and 500L in GMP[137] - The company has developed 45 analytical methods to test, monitor, and characterize its products, enhancing its manufacturing capabilities[137] - The company’s manufacturing strategy utilizes HEK293 transfection in a serum-free suspension bioreactor process, which is well-regarded by regulatory authorities[137] Market Competition and Challenges - The company faces competition from major pharmaceutical and biotechnology companies, as well as smaller firms, in the gene therapy and gene editing markets[140] - Third-party payors are increasingly reducing reimbursements for medical products, which could adversely affect sales and physician usage[192] - Environmental laws impact the company's operations, and compliance is necessary to avoid liabilities and fines[195] Disease Focus and Treatment Needs - The company is currently focused on monogenic diseases, utilizing gene therapy and gene editing approaches to address genetic abnormalities[56] - The company aims to transform the treatment paradigm for rare genetic diseases by delivering single-administration curative therapies[66] - The company intends to develop treatments for other inborn errors of metabolism in the liver as expertise in PKU treatment expands[115] - The incidence of PKU in the United States is estimated at one in 12,707, translating to approximately 350 new cases annually, with a total prevalence of 16,500[85] - Current treatment Kuvan generated worldwide sales of approximately $228 million in 2022, but it does not fully correct the underlying genetic disorder[87] - A five-year retrospective study confirmed that PKU patients on a low Phe diet had Phe levels well above the recommended threshold, highlighting the need for new treatments[86] Clinical Development Process - The regulatory approval process for the company's future product candidates is extensive and requires substantial time and financial resources[176] - The biological product candidate undergoes preclinical testing, including laboratory evaluations and animal studies, to assess safety and activity before human trials[178] - Phase I trials focus on safety testing in healthy subjects or patients for severe diseases, while Phase II evaluates efficacy and dosage in a limited patient population[179] - Phase III trials expand the patient population to establish the overall risk/benefit ratio and provide a basis for product labeling[179] - The FDA requires extensive monitoring and auditing of clinical activities, with annual progress reports and safety reports submitted for serious adverse events[180] - A Biologics License Application (BLA) must include results from all trials and manufacturing information, and must be submitted for FDA approval before commercial marketing[181] - The FDA reviews BLAs for safety, purity, and efficacy, and may refer applications to advisory committees for recommendations[182] - Changes to approved BLAs require submission of a new BLA or supplement, and may involve additional clinical data[182] - The FDA can withdraw product approval if compliance with requirements is not maintained or if safety issues arise post-marketing[182] - The Rare Pediatric Disease Priority Review Voucher program is authorized until September 30, 2024, allowing sponsors to receive a voucher for priority review of subsequent applications if their drug for a rare pediatric disease is approved[185]

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, 2022 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) (7 ...

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 March 31, 2022 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) D ...