Abstract
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Prasad is the permanent successor to Peter Marks, MD, MPH, who abruptly resigned in April after sharply criticizing the policies of Health and Human Services (HHS) Secretary Robert F. Kennedy Jr., a severe critic of the COVID-19 vaccines whose office has begun to overhaul the regulation of vaccines and drugs and has eliminated some 10,000 jobs across the FDA and other HHS agencies in recent weeks.
Prasad has publicly taken issue with several actions of Marks, who had overseen the approval of 22 gene therapies as CBER director. One is Marks’ leading role in shepherding agency review of vaccines funded through Operation Warp Speed, the first Trump administration’s initiative to catalyze development and deployment of COVID-19 vaccines and drugs.
Additionally, Prasad criticized Marks’ overruling of other FDA reviewers in allowing the agency to approve Sarepta Therapeutics’ Duchenne muscular dystrophy treatment Elevidys® (delandistrogene moxeparvovec-rokl), an adeno-associated virus vector-based gene therapy indicated to treat ambulatory and non-ambulatory people with Duchenne muscular dystrophy who are at least 4 years old and have a confirmed mutation in the dystrophin gene.
“Peter was a longtime proponent of lowering the bar for gene therapy. Against the advice of 3 FDA scientists on 2 occasions he granted both accelerated and regular approval to Sarepta’s Duchenne’s MD gene therapy. A therapy that has no evidence it helps boys [emphasis in original]. Sadly, now, at least 1 is dead,” Prasad wrote in March on his Substack feed, which he said will be inactive during his federal government service. “In short, Peter Marks was one of the most dangerous, pro-pharma regulators of the 21st century.” 1
Investor concerns about Prasad’s appointment leading to slower gene therapy reviews were reflected in declines to the stock prices of Sarepta and some higher profile gene and gene edited therapy developers, including UniQure, Rocket Pharmaceuticals, Beam Therapeutics, and Verve Therapeutics. 2
After Verve announced Phase I first human proof-of-concept data for in vivo base editing for its VERVE-101 focused on positive results such as low-density lipoprotein cholesterol reductions of up to 55% and up to 84% reductions in blood PCSK9 protein, Prasad highlighted Verve’s acknowledgment that two patients experienced serious adverse events, namely cardiovascular events in the context of severe underlying atherosclerotic cardiovascular disease.
“Americans don’t want that 2/10 pts with suspicious CV [cardiovascular] events. I agree with @venkmurthy that I would hold drug development of this product, and I surprised the stock has not collapsed more. Amazing what hype can do,” Prasad posted on X on November 16, 2023, amplifying a post by Venk Murthy, MD, PhD, a professor at the University of Michigan. 3
Verve later halted enrollment in the Heart-1 trial (NCT05398029) after a patient experienced a grade 3 serious adverse event (elevated liver enzyme and a low platelet count), though the company later said that the patient recovered and was discharged from hospital. The company pivoted its development effort toward another PCSK9-targeting heterozygous familial hypercholesterolemia candidate VERVE-102, which uses a different lipid nanoparticle delivery system than VERVE-101.
FDA Commissioner Martin Makary, MD, MPH, announced Prasad’s appointment on May 6. “With 500+ peer-reviewed publications and two books, Dr. Prasad brings the kind of scientific rigor, independence, and transparency we need at CBER—a significant step forward. Welcome aboard, Dr. Prasad.” 4
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LILLY INKS UP TO $1.4B+ AGREEMENT TO LICENSE SANGAMO AAV CAPSID
The agreement grants Lilly a worldwide exclusive license to use the STAC-BBB capsid for one initial target, with the right to add up to four additional targets after paying additional licensed target fees.
According to Sangamo, STAC-BBB has demonstrated potent blood–brain barrier penetration and neuronal transduction in nonhuman primates. Sangamo reasons that STAC-BBB can address long-standing challenges associated with delivering therapies to the central nervous system.
“We are pleased to be sharing STAC-BBB with Lilly to advance potential treatments for neurological diseases with significant unmet medical needs,” Sangamo CEO Sandy Macrae said. “This marks the third agreement with a pharmaceutical company since we announced the discovery of STAC-BBB in March 2024, and demonstrates the continued industry interest in our capsid delivery technology.” 5
The other two pharmas that have agreed to use STAC-BBB are Astellas and Genentech, a member of the Roche Group.
Sangamo has agreed to oversee completion of a technology transfer related to the STAC-BBB capsid. Lilly is responsible for all research, preclinical and clinical development, regulatory interactions, manufacturing, and global commercialization of any resulting gene therapy products, subject to certain specified reductions.
Lilly agreed to pay Sangamo $18 million upfront, up to $1.4 billion in additional licensed target fees and milestone payments across all five potential disease targets, as well as tiered royalties on potential net sales on treatments developed through the collaboration.
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FDA APPROVES ABEONA’S ZEVASKYN, CELL-BASED GENE THERAPY, FOR RDEB
The
Zevaskyn consists of a patient’s own keratinocytes, skin cells that are genetically modified to produce functional type VII collagen. Zevaskyn gene-modified cellular sheets are applied via surgery to a patient’s wounded areas, with a manufacturing run able to supply up to 12 credit card-sized sheets that can be joined together in a single application of the therapy to cover a large area or applied to multiple wounds.
“It’s almost like an application of tiles to cover a large area. And if you have a smaller area, one or two, or three sheets can cover that. So there’s flexibility of how many sheets you use, and it can be done in one sitting,” said Abeona president and CEO Vishwas (Vish) Seshadri, PhD. 6
Zevaskyn won FDA approval upon Abeona’s second filing of a biologics license application (BLA) for the therapy. The first BLA was rejected last year, through an FDA Complete Response Letter citing chemistry, manufacturing, and controls concerns related to Zevaskyn but not questioning its safety and efficacy as shown in clinical data.
The FDA based its approval on positive data from the Phase III VIITAL trial (NCT04227106) showing that across 43 large and chronic wounds treated with a single application of Zevaskyn, 81% of the wounds had healed by ≥50% after 6 months, compared with 16% of wounds receiving standard care. Fewer than 5% of patients experienced side effects such as procedural pain or itch.
In approving Zevaskyn, the FDA also granted Abeona a Rare Pediatric Disease Priority Review Voucher, which the company said it plans to sell.
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NUEVOCOR CLOSES $45M SERIES B FINANCING, PLANS TRIAL OF AAV GENE THERAPY
The Phase I trial—set to be launched in early 2026—will be designed as an open-label, multicenter, ascending single-dose study that will collect data from sites across the United States and Europe. Additionally, the company will establish an office in Paris, France, to support clinical development and bring innovation to patients globally from Nuevocor, which is based in Singapore and has U.S. offices in the Philadelphia suburb of Fort Washington, Pennsylvania.
NVC-001 and Nuevocor’s other pipeline assets are derived from the company’s PrOSIATM mechanobiology platform, which is designed to pinpoint the functional root cause of many cardiomyopathies by treating defects in shared disease pathways across multiple cardiomyopathies.
“We are delighted to secure this significant financing milestone to build a global company with such an experienced syndicate of international life science investors,” said Yann Chong Tan, PhD, Nuevocor’s CEO and cofounder. “The continued strong support from insiders and the addition of new investors will enable Nuevocor to continue its strong momentum and advance NVC-001 into the clinic.” 7
Kurma Partners and Angelini Ventures co-led Nuevocor’s Series B financing, with what the company called significant participation from existing investors EDBI, ClavystBio, and Boehringer Ingelheim Venture Fund, alongside Highlight Capital and SEEDS Capital.
In connection with the financing, Amanda Gett-Chaperot, PhD, partner with Kurma Partners, and Elia Stupka, PhD, managing director with Angelini Ventures, have been appointed to Nuevocor’s board of directors.
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CARLYLE GROUP, SK CAPITAL RAISE UPFRONT OFFER FOR BLUEBIRD BIO
The
“The amended offer price provides an alternative for stockholders who would prefer greater upfront cash consideration instead of the potential upside of the CVR,” Bluebird said in a statement. “The bluebird board of directors continues to believe that the transaction with Carlyle and SK Capital, as amended, represents the only viable option for stockholders to receive consideration for their shares.” 8
Bluebird’s board of directors unanimously approved the amended agreement and has recommended that all stockholders immediately tender their shares in support of the transaction. Should a majority not tender their shares, Bluebird was at significant risk of defaulting on its loan agreements with Hercules Capital, the company asserted, adding that stockholders would not likely receive any consideration for their shares in a bankruptcy or liquidation.
In connection with the amended agreement, the expiration date of the tender offer had been extended to expire at 1 min after 11:59 p.m., Eastern Time, on May 29.
The company added that any shares tendered for which no election is made will receive the original consideration.
In February, Bluebird entered into a definitive agreement to be acquired by funds managed by Carlyle and SK Capital, partnering with a team of veteran biotech executives led by David Meek. He is set to become CEO of the newly private Bluebird nearly 2 years after he left Mirati Therapeutics, where he spent 2 years as CEO before the company’s up-to-$5.8 billion acquisition by Bristol Myers Squibb, a deal completed in January 2024.
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FIRSTGENE TO ADVANCE HOVIONE’S AAV-LIKE PLATFORM TO TREAT HCC
Hovione has agreed to grant Firstgene an exclusive license to its proprietary novel targeted adeno-associated virus (AAV)-like particle technology for the treatment of HCC—a therapeutic indication developed by Firstgene—and will provide preclinical R&D services.
The virus-like particle technology will be engineered and functionalized for the specific carcinoma cell type using therapeutically validated AAVs, including a novel primer compound. Hovione has agreed to oversee molecular engineering, the supply of tailor-made virus-like particles, and the execution of proof-of-concept studies.
“The combination of Hovione’s expertise in complex chemistry and particle engineering and the commercialization expertise of Xlife Sciences positions Firstgene well to offer patients potential new safe and effective therapy options,” said Alexander Zink, Dr. rer. nat., Managing Director of Firstgene Life Sciences. 9
Hovione CEO Jean-Luc Herbeaux, PhD, added that his company was proud to advance the development of oncology treatments that address a critical unmet need for precision gene expression at the tissue level.
The value of the collaboration has not been disclosed.