Adrenoleukodystrophy

NATICK, Mass.--(BUSINESS WIRE)-- AffyImmune Therapeutics, Inc., a clinical-stage biopharmaceutical company committed to developing novel, first-in-class, affinity-tuned CAR T cell therapies, announced today the appointment of Matt Britz to Chief Executive Officer and Pete Gelinas as Senior Vice President of Chemistry, Manufacturing, and Controls. This press release features multimedia. View the full release here: Matt Britz, CEO, AffyImmune (Photo: Business Wire) “Matt has been an instrumental part of AffyImmune’s growth in the past two years,” said Simone Song, Chairperson of the Board of Directors, AffyImmune, and Founder and Senior Partner at ORI Capital. “AffyImmune is a company incubated by ORI Capital. Matt has executed a focused business strategy supported by insights generated from our big data platform ORIzon, which has led to promising early clinical results for AffyImmune’s lead program AIC100 in advanced thyroid cancer. Matt is well-suited to lead AffyImmune as CEO as the Company accelerates clinical development of AIC100 in thyroid cancer, and in other indications with high unmet medical need. I am also thrilled to welcome Pete to our leadership team at a critical time as we scale up CMC efforts to support our ongoing trial and potential indication expansion. Pete’s extensive BLA experience will be invaluable as AffyImmune moves to the next stage of growth.” “I’m honored to lead AffyImmune’s efforts in advancing affinity-tuned CAR T therapies, building upon the solid foundation we’ve established in the last few years,” said Britz. “Together, we're well-positioned to make significant advancements in addressing the challenges of solid cancers to improve patient outcomes." Matt Britz joined AffyImmune in June 2021 as Senior Vice President (SVP), Business Development and was promoted to Chief Operating Officer in January 2022. He brings 28 years of demonstrated experience in life sciences, and was previously SVP and Head of Business Development at Minerva Biotechnologies, where he led teams across corporate development, manufacturing, clinical operations and regulatory affairs. Prior to Minerva Biotechnologies, Matt held scientific, consulting, and business development positions at Merck, Pfizer, Equinox Group, Health Advances, and Envoy Therapeutics, and played a crucial role in the acquisition of Envoy Therapeutics by Takeda. Matt has expertise leading teams in the initial stages of drug discovery all the way through to development and commercialization. Matt has an MBA from Washington University St. Louis, and a BS in Chemical Engineering and Biology from MIT. Pete Gelinas brings over 15 years of extensive experience in Manufacturing and CMC Product Development to AffyImmune. Previously, he served as the Head of Manufacturing and Technical Operations at ElevateBio, where he led technology transfer, CMC strategy, and oversight of routine clinical manufacturing. During his tenure at bluebird bio, he led both the MSAT and CMC teams for Skysona, a treatment for Cerebral Adrenoleukodystrophy (CALD). He has executed numerous late-phase global technology transfer deals and played a pivotal role in driving the drug product process validation efforts critical to the successful Marketing Authorization Application (MAA) for Zynteglo. Peter has also held various positions at the Connell O’Reilly Cell Manipulation Core Facility (CMCF) at Dana Farber Cancer Institute (DFCI), where he contributed to the development and manufacturing of several gene and cell-based therapies. “I am thrilled to join AffyImmune at this next stage of growth, with the opportunity to contribute my expertise in manufacturing to advance this pipeline for patients with solid cancers,” said Gelinas. “I look forward to making a difference in the lives of patients and their families, and to deliver our life-changing therapies in areas with high unmet medical need.” About AffyImmune Therapeutics AffyImmune is a clinical-stage biopharmaceutical company committed to developing novel, first-in-class, affinity-tuned CAR T cell therapies to transform the lives of patients with cancer. The Company’s proprietary technology utilizing ICAM1/LFA-1 interaction can potentially address all critical CAR T challenges such as toxicity, antigen loss, T-cell trafficking and exhaustion, while also allowing in vivo tracking of the CAR T cells. Its lead candidate, AIC100, is a ICAM1-directed CAR T therapy being developed for patients with advanced thyroid cancer. AffyImmune is developing a focused pipeline of therapeutics targeting ICAM-1 in oncologic indications with high unmet medical need. AffyImmune is based in Natick, MA. For more information, visit .

With the loan, bluebird said its cash runway now extends to the first quarter of 2026. As bluebird bio works to make the most of three gene therapy launches, cash-flow concerns have been looming over the drugmaker.  Now, Hercules Capital has swooped in to offer a five-year loan—worth up to $175 million—that will significantly extend the company's runway. The first $75 million portion of the loan was drawn upon the deal closing, with two $25 million tranches available upon the achievement of certain commercial milestones, bluebird said in a Monday release.  A fourth $50 million pot “may be available at the sole discretion Hercules,” bluebird added. Excluding that tranche, the $125 million in loan funding should support the company's operations through the first quarter of 2026. “This financing underscores the value bluebird offers as a standalone gene therapy leader and meaningfully extends our runway, bolstering our ability to bring transformative treatments to patients and their families,” bluebird's chief financial officer, Chris Krawtschuk, said in the release. Bluebird in December scored FDA approval for its sickle cell disease (SCD) gene therapy Lyfgenia, albeit with a black-box warning. Bluebird set its wholesale acquisition cost at $3.1 million, about 40% higher than the price competitor Vertex put on its CRISPR Therapeutics-partnered SCD therapy Casgevy.  Still, bluebird CEO Andrew Obenshain has said there's much more to consider than just the starting price for the therapies. Bluebird has offered a “very innovative contract” for its drug that has been well received by payers, Obenshain said in January. Outside of Lyfgenia, bluebird also markets its one-time beta thalassemia treatment Zynteglo, which comes with a $2.8 million price tag. The company also sells cerebral adrenoleukodystrophy gene therapy Skysona, which is priced at $3 million. Those drugs won back-to-back approvals in 2022. Amid its regulatory advances, bluebird has sought new cash sources to support its commercial future. Other than the latest loan, the company has reached an agreement with Alterna Capital Solutions to hand over future revenues in exchange for upfront funding in a deal that covers a maximum credit of $100 million. It also announced in December that it was working with Goldman Sachs and J.P. Morgan Securities to sell $150 million in new shares.  Before all of those fundraising efforts, bluebird estimated that its cash and certain other assets could fund operations through the second quarter of 2024.

- Funding expected to extend the Company’s cash runway beyond the next 24 months - - First tranche of $75 million was drawn upon closing; the Company will be eligible to draw an additional $50 million subject to achievement of commercial milestones - SOMERVILLE, Mass.--(BUSINESS WIRE)-- bluebird bio, Inc. (NASDAQ: BLUE) (“bluebird bio” or the “Company”) today announced that it has entered into a $175 million five-year, term loan facility with Hercules Capital, Inc. (NYSE: HTGC) (“Hercules”). The transaction strengthens the Company’s balance sheet as it executes on the commercial launches for its three FDA approved gene therapies – LYFGENIA for sickle cell disease, ZYNTEGLO for beta-thalassemia and SKYSONA for cerebral adrenoleukodystrophy. The term loan facility provides for up to $175 million of term loans in aggregate, available in four tranches. Upon closing of the transaction, the first tranche of $75 million was drawn. Under the terms of the agreement, bluebird will be eligible to draw two additional tranches of $25 million each, subject to the achievement of commercial milestones. Based on launch trajectory and current business plans, and assuming three tranches totaling $125 million are executed, the transaction is expected to extend bluebird’s cash runway through the first quarter of 2026. A fourth tranche of up to $50 million may be available at the sole discretion of Hercules. During the first three years of the five-year term, the Company will be responsible for paying only the interest on any amounts borrowed; any outstanding balance as of April 1, 2027 will be amortized over the remaining life of the loan. “Since establishing bluebird as an independent gene therapy company in 2021, we have been focused on diligently deploying our capital and strengthening our balance sheet to further our mission,” said Chris Krawtschuk, chief financial officer, bluebird bio. “This financing underscores the value bluebird offers as a standalone gene therapy leader and meaningfully extends our runway, bolstering our ability to bring transformative treatments to patients and their families.” “Hercules is excited to partner with bluebird as they launch LYFGENIA and bring this transformational therapy to patients living with sickle cell disease,” said Michael Dutra, Managing Director and Senior Investment Officer at Hercules Capital. “We are proud to support bluebird’s mission of developing and commercializing treatments for severe genetic diseases. This financing should help support the availability of their novel gene therapies for patients,” added John Miotti, Principal at Hercules Capital. Additional details of the loan agreement will be filed with the Securities and Exchange Commission on a Current Report on Form 8-K. J. Wood Capital Advisors acted as sole financial advisor to the Company. Latham & Watkins LLP served as legal counsel to bluebird and DLA Piper served as legal counsel to Hercules. About bluebird bio, Inc. bluebird bio is pursuing curative gene therapies to give patients and their families more bluebird days. Founded in 2010, bluebird has been setting the standard for gene therapy for more than a decade—first as a scientific pioneer and now as a commercial leader. bluebird has an unrivaled track record in bringing the promise of gene therapy out of clinical studies and into the real-world setting, having secured FDA approvals for three therapies in under two years. Today, we are proving and scaling the commercial model for gene therapy and delivering innovative solutions for access to patients, providers, and payers. With a dedicated focus on severe genetic diseases, bluebird has the largest and deepest ex-vivo gene therapy data set in the field, with industry-leading programs for sickle cell disease, β-thalassemia and cerebral adrenoleukodystrophy. We custom design each of our therapies to address the underlying cause of disease and have developed in-depth and effective analytical methods to understand the safety of our lentiviral vector technologies and drive the field of gene therapy forward. bluebird continues to forge new paths as a standalone commercial gene therapy company, combining our real-world experience with a deep commitment to patient communities and a people-centric culture that attracts and grows a diverse flock of dedicated birds. For more information, visit bluebirdbio.com or follow us on social media at @bluebirdbio, LinkedIn, Instagram and YouTube. bluebird bio, ZYNTEGLO, SKYSONA and LYFGENIA are registered trademarks of bluebird bio, Inc. All rights reserved. Cautionary Note Regarding Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements, such as statements regarding the Company’s anticipated ability to draw future tranches under the loan agreement and the impact on the Company’s cash runway and statements regarding the Company’s plans and expectations for the launch trajectory of its approved gene therapies, the anticipated benefits of and activities under the loan agreement and the Company’s business strategy and plans. Such forward-looking statements are based on historical performance and current expectations and projections about our future financial results, goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, divert or change any of them in the next several years, that are difficult to predict, may be beyond our control and could cause our future financial results, goals, plans and objectives to differ materially from those expressed in, or implied by, the statements. No forward-looking statement can be guaranteed. Forward-looking statements in this press release should be evaluated together with the many risks and uncertainties that affect bluebird bio’s business, particularly those identified in the risk factors discussion in bluebird bio’s Annual Report on Form 10-K for the year ended December 31, 2022, as updated by our subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the Securities and Exchange Commission. These risks include, but are not limited to: delays and challenges in our commercialization and manufacturing of our products; the internal and external costs required for our ongoing and planned activities, and the resulting impact on expense and use of cash, has been, and may in the future be, higher than expected which has caused us, and may in the future cause us to use cash more quickly than we expect or change or curtail some of our plans or both; substantial doubt exists regarding our ability to continue as a going concern; our expectations as to expenses, cash usage and cash needs may prove not to be correct for other reasons such as changes in plans or actual events being different than our assumptions; the risk that the efficacy and safety results from our prior and ongoing clinical trials will not continue or be seen in additional patients treated with our product candidates; the risk of insertional oncogenic or other reportable events associated with lentiviral vector, drug product, or myeloablation, including the risk of hematologic malignancy; and the risk that any one or more of our products will not be successfully commercialized. The forward-looking statements included in this document are made only as of the date of this document and except as otherwise required by applicable law, bluebird bio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, changed circumstances or otherwise.