Financial Position - As of September 30, 2020, the company had cash, cash equivalents, and marketable securities of approximately $1.44 billion[147]. - The company expects its current cash position to fund operations into 2023, assuming continued focus on expense management and exploration of additional funding sources[153]. - As of September 30, 2020, the company has $120.0 million in future contingent cash payments related to the acquisition of Precision Genome Engineering, with $20.1 million tied to clinical milestones and $99.9 million to commercial milestones[171]. Revenue Generation - The company has not generated any revenue from product sales to date, relying instead on collaboration arrangements and research fees[154]. - A one-time up-front payment of $200 million was received from BMS as part of an amended agreement related to ide-cel and bb21217[153]. - The company recognized $26.9 million in revenue from LentiGlobin for the three months ended September 30, 2020, compared to $26.5 million for the same period in 2019, indicating a slight increase[165]. - Total revenue for the three months ended September 30, 2020, was $19.3 million, an increase of $10.4 million from $8.9 million for the same period in 2019, primarily due to increased ide-cel license and manufacturing services revenue[176]. - Total revenue for the nine months ended September 30, 2020, was $240.0 million, an increase of $205.3 million from $34.7 million for the same period in 2019, largely due to a cumulative catch-up adjustment related to a contract modification[181]. - License revenue has historically been generated from out-license agreements, with potential future milestone payments and royalties recognized as revenue[158]. Expenses and Losses - The net loss for the three and nine months ended September 30, 2020, was $194.7 million and $418.8 million, respectively, with an accumulated deficit of $2.70 billion[147]. - The company expects to incur significant expenses and operating losses for at least the next several years due to ongoing clinical studies and commercialization efforts[147]. - The company anticipates an increase in selling, general and administrative expenses as it executes commercial launch plans for ZYNTEGLO in Europe and prepares for activities in the United States[167]. - Selling, general and administrative expenses for the three months ended September 30, 2020, were $68.0 million, an increase of $1.8 million from $66.3 million for the same period in 2019, primarily driven by increased consulting fees[178]. - Selling, general and administrative expenses for the nine months ended September 30, 2020, were $209.9 million, an increase of $14.8 million from $195.2 million for the same period in 2019, primarily due to increased employee compensation and IT-related costs[184]. Research and Development - The company reported total direct research and development expenses of $84.5 million for the three months ended September 30, 2020, compared to $93.5 million for the same period in 2019, reflecting a decrease of approximately 9.5%[165]. - Total research and development expenses for the nine months ended September 30, 2020, were $450.9 million, an increase of approximately 7.2% compared to $420.6 million for the same period in 2019[165]. - The company plans to continue investing in research and development for product candidates including beti-cel, eli-cel, and LentiGlobin, focusing on severe genetic diseases and oncology[163]. - Research and development expenses include costs associated with multiple ongoing clinical studies, such as the Northstar-2 Study and the KarMMa study, which are critical for advancing product candidates[163]. - The company has incurred research and development expenses related to various studies, including a total of $140.4 million for the three months ended September 30, 2020, compared to $151.4 million for the same period in 2019[165]. - Research and development expenses for the nine months ended September 30, 2020, were $450.9 million, an increase of $30.3 million from $420.6 million for the same period in 2019, driven by increased employee compensation and clinical trial costs[182]. Impact of COVID-19 - The COVID-19 pandemic has caused disruptions in clinical trials and manufacturing, impacting the company's operations and timelines[150]. - The company has prioritized key research and development programs in response to the COVID-19 pandemic, adjusting its future cost structure accordingly[152]. Market Risks - The company is exposed to market risk related to interest rates, with a hypothetical decline of approximately $6.8 million in the fair value of interest-sensitive marketable securities if rates increase by 100 basis points[195].

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 __________________________________________________________________ Title of each class Trading Symbol(s) Name of each exchange on which registered Common Stock, $0.01 par value per share BLUE The NASDAQ Stock Market LLC 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, 2020 ...

Financial Data and Key Metrics Changes - The company has made significant adjustments to its operating plan, removing over $500 million from its operating budget through mid-2022, which extends its financial runway while maintaining core programs [10][20][24] - The revised operating plan aims to balance innovation with financial sustainability, focusing on delivering core programs by 2022 and securing non-dilutive funding [20][21] Business Line Data and Key Metrics Changes - The company submitted a US Biologics License Application (BLA) for ide-cel for late-stage multiple myeloma treatment, with plans for a data presentation at the virtual ASCO meeting [8][18] - For sickle cell disease, the company reached alignment with the FDA on an accelerated approval path for LentiGlobin, planning to file for approval in the second half of 2021 [9][17] Market Data and Key Metrics Changes - The company is actively engaging with European authorities for the sickle cell opportunity, aiming to ensure access to data and finalize requirements for regulatory approval [66] - The company anticipates the first commercial patients for ZYNTEGLO in Germany later this year, with ongoing reimbursement discussions in other EU markets [70] Company Strategy and Development Direction - The company is focused on delivering transformative products while maintaining a strong operational foundation, emphasizing the importance of culture and innovation [11][20] - The revised collaboration with Bristol-Myers Squibb (BMS) includes a $200 million non-dilutive funding agreement, enhancing operational flexibility and aligning manufacturing responsibilities [22] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's ability to navigate the challenges posed by the COVID-19 pandemic, highlighting the resilience and adaptability of the team [7][12] - The company remains committed to its long-term vision while adapting to the current economic landscape, ensuring that core values and innovation are preserved [11][20] Other Important Information - The company plans to present updated clinical data for Lenti-D for CALD at a medical meeting later this year, with regulatory filings in the EU planned for this year and in the US next year [19] - The company is prioritizing the core four late-stage programs while optimizing operational resources to ensure sustainability [20][24] Q&A Session Summary Question: What does "general agreement" mean regarding the sickle cell path? - Management clarified that "general agreement" refers to a legal categorization and does not change the agreements with the FDA, confirming that the filing will be based on HGB-206 data, not HGB-210 [28][29] Question: How does the $200 million correlate with the ex-US opportunity for bb2121? - Management expressed confidence that the deal reflects significant value and aligns operational responsibilities, ensuring a sensible split of resources [32][34] Question: What are the gating items on manufacturing related to FDA for sickle cell? - Management indicated that clinical alignment and manufacturing transitions are well underway, with ongoing discussions to ensure readiness for regulatory timelines [48][50] Question: Why is HGB-210 necessary as a post-approval confirmatory study? - Management explained that HGB-210 serves as a classic post-approval confirmatory trial to provide additional follow-up data for full approval [58] Question: What is the expected timeline for the sickle cell filing? - Management anticipates submitting the BLA in the second half of 2021, with a focus on complete resolution of severe vaso-occlusive events as the primary endpoint [17][55]

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, 2020 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: ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 ___________________________________________________________________________ FORM 10-K ___________________________________________________________________________ (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2019 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission ...