September 30, 2022 / Clinical Trials

Solid Biosciences Acquires AavantiBio & Shares Update on Duchenne Gene Therapy Pipeline Programs

Today Solid Biosciences shared an update with the Duchenne community regarding their strategic development. Solid announced they have acquired AavantiBio, a gene therapy company with a focus on rare cardiomyopathies and Friedeich’s ataxia. While this will expand Solid’s pipeline for developing therapeutics for other diseases, they are still dedicated to bringing forward treatments for Duchenne. With the acquisition AavantiBio’s CEO will step into the CEO role for Solid Biosciences, Ilan Ganot, co-founder of Solid, will remain on Solid’s Board of Directors and serve as a strategic advisor.

In the announcement they also shared their decision to prioritize SGT-003, their next generation gene transfer product. SGT-003 utilizes a novel AAV, that in pre-clinical models has shown higher expression and more targeted bio-distribution, along with their nNos differentiated microdystrophin transgene (preclinical data on SGT-003 was shared earlier this year). They anticipate an IND submission for SGT-003 mid-2023 with dosing of patients in a clinical trial by late 2023.  Solid will pause activities for its first-generation gene transfer therapy candidate SGT-001, intending to complete currently ongoing SGT-001 preclinical and manufacturing activities.

As more information about SGT-003 is made available we will be sure to update the community. For more information please see the community letter from Solid below.

Read the Community Letter

Letter to the Duchenne Community: Strategic Update

Dear Duchenne Community,

In a press release issued this morning, we announced Solid’s entry into a definitive merger agreement to acquire AavantiBio, Inc., to create a genetic medicines company focused on neuromuscular and cardiac rare diseases, maintaining a strong focus on Duchenne. We want to share more context with you and what this means for the future of Solid and our programs.

Acquisition of AavantiBio

Upon closing of the merger, the combined company, which will operate as Solid Biosciences, will bring together exceptional scientists and life science leaders who are committed to patients with Duchenne and other, adjacent rare diseases. While Solid’s pipeline will include other neuromuscular and cardiac disease indications, we will carry forward our steadfast dedication to the Duchenne community and to advancing meaningful therapies. Among new leaders that will join Solid, Bo Cumbo, current President and Chief Executive Officer (CEO) of AavantiBio, will assume the role of President and CEO of Solid Biosciences at closing, succeeding Solid’s Co-Founder, President and CEO, Ilan Ganot, who will remain on Solid’s Board of Directors and as a strategic advisor to the CEO. A seasoned biotech executive, Bo shares Solid’s commitment to patients, and has gained valuable experience bringing drugs through the clinic to commercialization, and of all diseases, particularly in Duchenne. The acquisition is expected to close by the end of 2022, subject to approval by Solid’s stockholders and satisfaction of other customary closing conditions.

Many of you have met Ilan and Annie Ganot, Solid’s President and CEO and Head of Patient Advocacy, respectively. Both have been deeply involved with the Duchenne community since their son Eytani’s diagnosis in 2012. Over the past 10 years, Ilan has guided Solid Biosciences, navigating through challenges, growth and milestones, and ultimately driving Solid’s strategy toward our mission to advance meaningful treatments for patients with Duchenne.

SGT-003 and SGT-001

Today, we also announced that we have made the strategic decision to prioritize SGT-003, our next-generation adeno-associated virus (AAV) gene transfer therapy candidate that utilizes a novel, next generation capsid (AAV-SLB101), to deliver Solid’s proprietary and differentiated nNOS microdystrophin protein. We also released new SGT-003 non-clinical data which reinforced previous comparative analyses that have demonstrated increased microdystrophin expression using the novel muscle-tropic capsid AAV-SLB101 compared to AAV9. Solid remains on track for an anticipated IND submission for SGT-003 in mid-2023 with patient dosing to begin in late-2023, pending IND acceptance.

We also announced Solid will be pausing activities for its first-generation gene transfer therapy candidate SGT-001. Solid intends to complete currently ongoing SGT-001 preclinical and manufacturing activities in order to be in a position to reactive the program in the future, if desired.

Solid has continually evaluated the two programs, and believes that this is the right opportunity to put all of Solid’s Duchenne efforts behind what we believe to be a best-in-class gene therapy candidate. We will continue to follow and report data from patients who were treated in IGNITE DMD, the Phase I/II trial of SGT-001 as we believe it will continue to build on the benefit Solid’s nNOS microdystrophin can offer to patients.

We would like to express our immense gratitude for the patients and families who participated in the IGNITE DMD clinical trial, as well as all those who choose to participate in clinical trials. Your bravery and willingness provide the important foundation for advancing science and needed therapies for Duchenne.

Solid’s Exciting Future

We are excited about the potential Solid has to bring meaningful treatments to patients with Duchenne as well as other rare diseases. With a strong leadership team, dedicated scientists and rigorous science being conducted every day, Solid will be well-resourced and well-positioned for our future.

As always, we appreciate your continued support and look forward to building on our strong relationships and continuing to nurture our engagement with the Duchenne community.


Your Solid Biosciences Team

Upcoming Community Presentations

  • Action Duchenne International Conference 2022: Nov. 11-12, Leicestershire, U.K.

Read the full community letter.

Read the Press Release:

Solid Biosciences Announces Acquisition of AavantiBio and Concurrent $75 Million Private Placement
September 30, 2022 at 7:00 AM EDT

CHARLESTOWN, Mass. and CAMBRIDGE, Mass., Sept. 30, 2022 (GLOBE NEWSWIRE) — Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy (Duchenne), and AavantiBio, Inc., a privately-held gene therapy company focused on transforming the lives of patients with Friedreich’s ataxia and rare cardiomyopathies, today announced that the companies have entered into a definitive merger agreement whereby Solid will acquire AavantiBio, including its pipeline assets and net cash. The combined company will focus on advancing a portfolio of neuromuscular and cardiac programs, led by SGT-003, a differentiated gene transfer candidate, for the treatment of Duchenne. Additional pipeline programs include AVB-202, a gene transfer candidate for the treatment of Friedreich’s ataxia, AVB-401 for BAG3 mediated dilated cardiomyopathy, and additional assets for the treatment of undisclosed cardiac diseases. Following approval by Solid stockholders, the combined company will operate as Solid Biosciences, will trade on Nasdaq under the ticker symbol “SLDB” and Bo Cumbo, the current Chief Executive Officer of AavantiBio, will assume the role of President and CEO of Solid Biosciences.

In support of the acquisition, Solid announced it has entered into a securities purchase agreement with a select group of institutional investors and accredited investors for a $75 million private placement that is expected to close concurrently with the closing of the merger. The private placement is being led by existing investors Perceptive Advisors, LLC, RA Capital Management and Bain Capital Life Sciences, and other new and existing investors participating in the private placement include CaaS Capital Management, Invus, Laurion Capital Management and Pura Vida Investments.

Immediately following the closing of the merger and financing, the total cash and investments of the combined company is expected to be approximately $215 million. Solid expects this will be sufficient to fund the combined company’s planned operating expenses and capital expenditure requirements into 2025 and enable the potential attainment of key milestones for the combined company’s lead programs. The merger and private placement are expected to close in the fourth quarter of 2022, subject to customary closing conditions.

“I created Solid with my wife, Annie, and our co-founders nearly ten years ago, to bring meaningful treatment options to patients and families who, like ours, live with the devastating consequences of Duchenne muscular dystrophy,” said Ilan Ganot, President, Chief Executive Officer and Co-Founder of Solid Biosciences. “This acquisition provides exciting opportunities to bring our potentially best-in-class Duchenne gene transfer candidate, SGT-003, to patients and to expand our portfolio with innovative gene therapies designed to address significant unmet need in additional, adjacent rare disease indications. Bo Cumbo will assume the role of President and CEO and is a seasoned biotech executive with extensive expertise in bringing products through development and commercialization. I look forward to helping Bo with the leadership transition following the acquisition, and I am grateful to the investors who continue to support Solid’s critical efforts.”

“I am excited for the opportunity to lead Solid Biosciences and to help build a leading genetic medicine company with a focus on neuromuscular and cardiac rare diseases,” said Bo Cumbo, President and Chief Executive Officer of AavantiBio. “The Solid team has done incredible work in advancing Duchenne gene therapy, and its commitment to the Duchenne community aligns with AavantiBio’s patient-centric mission of bringing new therapies that can positively improve the quality of life of rare disease patients and their families. I also look forward to advancing our library of next generation cardiac and skeletal muscle capsids to extend our opportunities to bring treatment options to more people in need.”

About The Combined Company Pipeline
The combined company will have a diversified pipeline across neuromuscular and cardiac diseases with indications Solid believes are characterized by high unmet need, clear mechanistic rationale and significant market opportunities.

Duchenne Muscular Dystrophy
Duchenne is a fatal neuromuscular disease caused by mutations in the gene encoding dystrophin that lead to the absence or near-absence of functional dystrophin protein. Today, Solid announced it has made the strategic decision to prioritize SGT-003, its next-generation adeno-associated virus (AAV) gene transfer therapy candidate that utilizes a rationally designed, novel muscle-tropic AAV capsid (AAV-SLB101), to deliver Solid’s proprietary and differentiated neuronal nitric oxide synthase (nNOS) microdystrophin protein. Solid also announced it will be pausing activities for its first-generation gene transfer therapy candidate SGT-001. Solid intends to complete currently ongoing SGT-001 preclinical and manufacturing activities in order to be in a position to reactivate the program in the future, if desired.

Development activities for SGT-003 continue and Solid anticipates submitting an investigational new drug application (IND) for SGT-003 in mid-2023 and, subject to IND clearance, initiating patient dosing in late-2023.

Friedreich’s Ataxia (FA)
FA is a rare inherited neuromuscular disease that causes progressive nervous system damage and movement problems. AVB-202, AavantiBio’s lead AAV gene transfer therapy candidate in preclinical development, utilizes a dual route of administration to more rigorously target disease pathology. Preclinical data from three animal models, including mouse and nonhuman primate, supported preclinical proof of concept. Solid is anticipating an IND submission for AVB-202 in the second half of 2024.

BAG3 Mediated Dilated Cardiomyopathy (BAG3) and Undisclosed Cardiac Diseases
BAG3 is a rare cardiac disease and is characterized by mutations in the BAG3 gene. Sufficient levels of functional BAG3 are required for healthy cardiac function. AavantiBio is currently developing AVB-401, a preclinical-stage product candidate, for the treatment of BAG3. Following the acquisition of AavantiBio, Solid will continue to develop AVB-401, as well as two early-stage cardiac programs initially developed by AavantiBio as part of its pipeline activities.

Next generation AAV capsid libraries for targeted tissue delivery
Solid will continue development programs for novel AAV capsids that are expected to enhance select tissue tropism for cardiac and skeletal muscle as well as reduce liver targeting. The first capsid screened in the Solid-based skeletal muscle library is AAV-SLB101, which is used in SGT-003 and has demonstrated increased expression and biodistribution compared with AAV9 in multiple preclinical studies. We believe this capsid has the potential to be used in other neuromuscular and cardiac indications. In addition, Solid plans to continue current AavantiBio efforts to develop novel AAV cardiac-targeted capsids that enhance select tissue tropism and reduce liver targeting.

About the Proposed Transactions, Management and Organization
Management and Organization
Following the closing of the transactions, Bo Cumbo, will be the President and Chief Executive Officer of the combined company, which will continue to operate as Solid Biosciences. The executive leadership team of the combined company will also include Stephen DiPalma, Interim Chief Financial Officer, Carl Morris, Ph.D., Chief Scientific Officer for Neuromuscular Diseases, Jenny Marlowe, Ph.D., Chief Scientific Officer for Friedreich’s Ataxia and Cardiac Pipeline, Roxana Donisa Dreghici, M.D., Head of Clinical Development, Jessie Hanrahan, Ph.D., Chief Regulatory Officer, Paul Herzich, Chief Technology Officer and Ty Howton, Chief Administrative Officer. Upon the closing of the transactions, Solid will add Bo Cumbo and Adam Koppel, M.D., Ph.D., managing director at Bain Capital Life Sciences to the board of directors. Ilan Ganot will continue to serve on Solid’s board of directors.

Transaction Details
Pre-combination equity holders of Solid are expected to own approximately 85% of the combined company and pre-combination equity holders of AavantiBio are expected to own approximately 15% of the combined company, subject to certain adjustments set forth in the merger agreement and in each case before giving effect to the private placement. The merger agreement has been unanimously approved by the Board of Directors of each company, and by the stockholders of AavantiBio.

In the private placement, Solid agreed to sell 159,574,463 shares of common stock at a price of $0.47 per share, and upon the closing of the private placement, will receive gross proceeds of $75 million.

The securities to be sold in the private placement will not be registered under the Securities Act of 1933, as amended (Securities Act), or any state or other applicable jurisdiction’s securities laws, and may not be offered or sold in the United States absent registration or an applicable exemption from the registration requirements of the Securities Act and applicable state or other jurisdictions’ securities laws. Solid has agreed to file a registration statement with the U.S. Securities and Exchange Commission (SEC) registering the resale of the shares of common stock issued in the acquisition and in the private placement no later than the 60th day after the closing of the private placement.

The acquisition and private placement are expected to close before the end of 2022 with the private placement closing as of immediately following the acquisition, subject to approval by the stockholders of Solid and the satisfaction of other customary closing conditions.

BofA Securities acted as the sole placement agent for the private placement made to institutional investors. Wilmer Cutler Pickering Hale and Dorr LLP is acting as legal counsel to Solid Biosciences. Sidley Austin LLP is acting as legal counsel to AavantiBio.

Conference Call Information
The companies will host a conference call today, September 30, 2022, at 8:00 a.m. ET, to discuss the transactions. A live webcast of the call will be available on Solid’s website at under the “News & Events” tab in the Investor Relations section, or by clicking here. Participants may also access the call by dialing 877-407-2991 (domestic) or 201-389-0925 (international) five minutes prior to the start of the call and providing the Conference ID 13733092.

The archived webcast will be available in the “News and Events” section of Solid’s website.

About Solid Biosciences Inc. 
Solid Biosciences is a life sciences company focused on advancing transformative treatments to improve the lives of patients living with Duchenne. Disease-focused and founded by a family directly impacted by Duchenne, our mandate is simple yet comprehensive – work to address the disease at its core by correcting the underlying mutation that causes Duchenne with our gene therapy candidate and SGT-003. For more information, please visit

About SGT-003
SGT-003 is Solid’s next-generation AAV gene transfer therapy candidate that utilizes a rationally designed, novel muscle-tropic AAV capsid, called AAV-SLB101, to deliver Solid’s proprietary and differentiated nNOS microdystrophin for the treatment of Duchenne. AAV-SLB101 has demonstrated enhanced muscle biodistribution and transgene expression, as well as reduced liver tropism, compared with AAV9 in in vivo mouse models and, utilizing a reporter transgene, non-human primate in vivo models. SGT-003 has correspondingly demonstrated higher levels of microdystrophin expression in vivo in the mdx mouse model of Duchenne and in vitro in human Duchenne cell lines. Solid is targeting an IND submission for SGT-003 in mid-2023.

About AavantiBio, Inc.
AavantiBio is a gene therapy company focused on advancing innovative gene therapies in areas of high unmet medical need, including a lead program in Friedreich’s ataxia, a rare inherited genetic disease that causes cardiac and central nervous system dysfunction, and programs in dilated or hypertrophic cardiomyopathies, and a next generation cardiac capsid library. The company benefits from strategic partnerships with the University of Florida’s Powell Gene Therapy Center and the MDA Care Center at UF Health where AavantiBio’s co-founders and renowned gene therapy researchers Barry Byrne, M.D., Ph.D. and Manuela Corti, P.T., Ph.D. maintain their research and clinical practices. Learn more at

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