Taysha Gene Therapies, Inc.

In explaining his departure, Marks pointed to an “unprecedented assault on scientific truth” in the United States and called out HHS secretary Robert F. Kennedy Jr. directly for promoting misinformation about the safety of vaccines. As the FDA wavers from cuts, changes and departures under the second Trump administration, another vital figure of the agency is heading for the exit.Friday, Peter Marks, M.D., Ph.D., revealed that he will relinquish his role as director of the FDA’s Center for Biologics Evaluation and Research (CBER) on April 5.“It is with a heavy heart that I have decided to resign from FDA,” Marks said in a resignation letter addressed to the agency’s acting commissioner Sara Brenner.In explaining his departure, Marks pointed to an “unprecedented assault on scientific truth” in the United States and called out Health and Human Services (HHS) Secretary Robert F. Kennedy Jr. directly for promoting misinformation about the safety of FDA-approved vaccines.The Wall Street Journal, citing people close to the matter, noted that an unnamed HHS official gave the director the choice to either resign from his role or face termination.“If Peter Marks does not want to get behind restoring science to its golden standard and promoting radical transparency, then he has no place at FDA under the strong leadership of Secretary Kennedy,” the HHS told the publication. Marks began his role as CBER director in 2016 and has played a key role in the evaluation and approval of vaccines, blood products and certain biologics in the U.S., including cell and gene therapies.During his tenure, Marks made a name for himself as an advocate for the FDA’s accelerated approval pathway for rare diseases, especially in conjunction with gene therapies. He also had a major hand in 2020’s Operation Warp Speed effort, which saw the biopharma industry join forces with the federal government to accelerate the development of COVID-19 vaccines during the height of the pandemic.In his resignation letter, Marks did not mince words about the rise of vaccine skepticism in the U.S., warning about “[e]fforts currently being advanced by some on the adverse health effects of vaccination.”Marks flagged an ongoing measles outbreak across multiple U.S. states—which has hit Texas the hardest—as a sobering reminder of “what happens when confidence in well-established science underlying public health and well-being is undermined.”“I was willing to work to address the Secretary’s concerns regarding vaccine safety and transparency by hearing from the public and implementing a variety of different public meetings and engagements with the National Academy of Sciences, Engineering, and Medicines,” Marks said in his letter, referring to Kennedy by his HHS title.“However, it has become clear that truth and transparency are not desired by the Secretary, but rather he wishes subservient confirmation of his misinformation and lies,” the outgoing CBER director continued.Kennedy proved a controversial nominee to lead the HHS from the start, thanks to his long-held and oft-publicized views endorsing vaccine skepticism and questionable science.Despite pledging not to meddle with vaccines during his confirmation hearing in January, Kennedy swiftly reversed course after taking up office at the HHS the following month, announcing an investigation into potential links between chronic disease rates and factors like childhood vaccination schedules, mental health medications and ultra-processed foods.Under Kennedy’s stewardship, two vaccine advisory meetings, one at the Centers for Disease Control and Prevention (CDC) and the other the FDA, have also been put on hold, while the HHS chief said last week on News Nation that he aims to create an agency within the CDC to study vaccine injuries. Marks' exit echoesDespite apparent pushback from within the FDA and HHS more broadly, the biopharma industry was swift to condemn the circumstances that seem to have fueled Marks’ exit.“We are deeply concerned that the loss of experienced leadership at the FDA will erode scientific standards and broadly impact the development of new, transformative therapies to fight diseases for the American people,” John Crowley, CEO of the trade organization the Biotechnology Innovation Organization (BIO), said in a statement Saturday.Meanwhile, the Alliance for Regenerative Medicine (ARM), reflecting on Marks’ influence in the cell and gene therapy space, said in its own statement that maintaining current regulatory standards for the field is “critical to the U.S.’ global leadership position.”“As the Administration and FDA consider resource allocation and restructuring decisions, we strongly encourage leaders to build on recent reform and modernization efforts and enable a culture that attracts, retains, and develops top scientific talent,” ARM continued.Several industry watchers echoed ARM’s concerns about the effect Marks’ exit could have on advanced therapeutics and the biotech outfits behind them. Analysts at William Blair wrote in a note Friday that the move is likely to weigh heavily on smaller and midsized companies working in vaccines, gene editing and cell and gene therapy.The market is currently reflecting the news as well, with shares from vaccine and gene therapy players like Moderna, Pfizer, Novavax, Taysha Gene Therapies, Solid Biosciences and Sarepta Therapeutics all down in premarket trading Monday, according to Reuters. Still, the situation may not be as dire as it seems, at least as far as advanced therapeutics for rare diseases are concerned.As it stands, there doesn’t appear to be any new regulatory risk for companies already working with the FDA on expedited pathways for rare diseases, analysts at Jefferies wrote in a series of notes over the weekend. The team said on Saturday that they hadn’t picked up on any timing delays for companies pursuing accelerated approvals and figure whoever steps up to captain CBER next is unlikely to dismantle the department’s existing rare disease programs. What's next at the FDA?The loss of Marks at the FDA comes at a tumultuous time for the regulator and for U.S. federal health agencies in general.While the U.S.’ chief drug regulator received a new commissioner last week in Johns Hopkins surgeon Marty Makary, M.D., the agency has also suffered several other high-profile departures since the start of the year.Most notably, Patrizia Cavazzoni, M.D., who served as head of the FDA’s Center for Drug Evaluation and Research (CDER), left the agency in mid-January. Cavazzoni, who had been with the FDA for seven years, has since been tapped to fill in as Pfizer’s new chief medical officer.More recently, the FDA’s chief counsel Hilary Perkins confirmed her resignation in mid-March, while other high-ranking agency staffers, such as Paul Kluetz, M.D., and Marc Theoret, M.D.—both of whom are deputy directors of the regulator’s Oncology Center of Excellence—are eyeing the exit, according to the William Blair team’s weekend note. The agency is undergoing a wider headcount reduction, too. Last week, the Trump administration announced plans to cut 3,500 full-time FDA staffers and another 1,200 NIH workers as part of an overall quest to eliminate 10,000 jobs across the HHS.As for who might replace Marks at CBER, the question remains open-ended for now, though there’s “clearly a risk that the candidate’s views will be in line with Secretary Kennedy’s on vaccines,” the William Blair analysts said. Even still, the team espoused “some hope” that Makary will make good on his new position as commissioner to “ensure a suitable candidate.” 

FDA The stock market—and biotech insiders—reacted negatively to the allegedly forced resignation of CBER Director Peter Marks, who said RFK Jr. does not seek “truth and transparency” but rather “subservient confirmation of his misinformation and lies.” Shares of several biotechs tumbled Monday after news broke this weekend that Peter Marks, head of the FDA’s Center for Biologics Evaluation and Research, had resigned. Marks’ “abrupt resignation” equals a “new wave of uncertainty at FDA,” BMO Capital Markets said in a Monday investor note. Marks, who has helmed the CBER division since 2016, has been a staunch supporter of vaccines, accelerated approval and regulatory flexibility for gene therapies for rare diseases . While Marks had made clear his intention to stay in his role under the new Trump administration, he was forced Friday to choose between resignation and being fired, according to several media outlets . In his resignation letter , Marks said he was “willing to work” to address the concerns expressed by Health and Human Services (HHS) Secretary Robert F. Kennedy Jr. about the safety of vaccinations. However, “it has become clear that truth and transparency are not desired by the secretary, but rather he wishes subservient confirmation of his misinformation and lies,” he wrote. Kennedy, who was confirmed as HHS secretary in February, has a long history of vocal vaccine mistrust—something that was broadly raised during his Senate confirmation hearings . “We view this departure as a significant negative for the biopharma and biotech sectors, as FDA’s independence rooted in sound scientific rigor is critical for their efficient functioning,” the BMO analysts added in their note. Marks’ departure follows that of several other prominent FDA leaders, including Patrizia Cavazzoni, who left her post as head of the agency’s Center for Drug Evaluation and Research (CDER) in January, resurfacing as Pfizer’s new chief medical officer last month. According to a Monday investor note by William Blair, Oncology Center of Excellence (OCE) deputy directors Paul Kluetz and Marc Theoret also plan to leave the agency “imminently” and “there are concerns that [OCE Director] Richard Pazdur . . . may also be at risk of leaving.” As was indicated by Marks in his resignation letter, the main points of contention between him and Kennedy were vaccine safety and transparency , the latter of which has also been a concern for the FDA and biotech companies since President Donald Trump took office in January. In reference to the job cuts enacted at the FDA in February shortly after Kennedy’s confirmation, Ovid Therapeutics CEO Jeremy Levin told BioSpace , “The extent and the rapidity of the actions taken has been remarkable, and that has perhaps led to, in many cases, the concern that there is no understanding of exactly why they’re being done.” In a LinkedIn post this weekend, Levin expressed “grave concern” over Marks’ resignation. “His resignation is far more than the departure of a highly respected figure. It is a stark and unequivocal warning,” Levin wrote. “In this letter—especially in the final paragraphs—Dr. Marks makes clear that the FDA, long held as the gold standard in global regulatory science, is now under threat from political interference and the systematic distortion of scientific truth.” The biotech stock market appeared to heed this warning, as gene therapy– and vaccine-focused companies including Taysha Gene Therapies, Solid Biosciences, Sarepta and Novavax, all fell between 5% and 10% before the bell on Monday, Reuters reported . “Our industry conversations over the weekend suggest the resignation of Dr. Peter Marks . . . was a negative surprise, and we expect companies with vaccine businesses to be volatile this week as investors digest this update,” Truist Securities wrote in a note Monday morning. The analysts also expressed concern that “a CBER yes-person may expand the influence of RFK Jr’s vaccine rhetoric.” Marks’ last day at CBER will be this Saturday, April 5.

DALLAS, Dec. 06, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today announced that, on December 2, 2024, the Compensation Committee of Taysha's Board of Directors granted one new employee an option to purchase 152,000 shares of the Company's common stock in connection with their employment. The stock option was granted under the Taysha Gene Therapies, Inc. 2023 Inducement Plan as an inducement material to the individual entering employment with Taysha in accordance with Nasdaq Listing Rule 5635(c)(4). The stock option has an exercise price of $3.08 which is equal to the closing price of Taysha's common stock on the date of grant. The stock option has a 10-year term and will vest over four years, with 25% of the option vesting on the first anniversary of the vesting commencement date and the remaining 75% of the option vesting in equal monthly installments over the 36 months thereafter. Vesting of the stock option is subject to such employee's continued service to Taysha on each vesting date. About Taysha Gene TherapiesTaysha Gene Therapies (Nasdaq: TSHA) is a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system. Its lead clinical program TSHA-102 is in development for Rett syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies that address the genetic root cause of the disease. With a singular focus on developing transformative medicines, Taysha aims to address severe unmet medical needs and dramatically improve the lives of patients and their caregivers. The Company’s management team has proven experience in gene therapy development and commercialization. Taysha leverages this experience, its manufacturing process and a clinically and commercially proven AAV9 capsid in an effort to rapidly translate treatments from bench to bedside. For more information, please visit http://www.tayshagtx.com. Company Contact:Hayleigh Collins Director, Head of Corporate Communications and Investor RelationsTaysha Gene Therapies, Inc.hcollins@tayshagtx.com Media Contact:Carolyn HawleyInizio EvokeCarolyn.hawley@inizioevoke.com