Life Sciences Voice

Gilead Secures Arenavirus Vaccine Programs from Hookipa for $10 Million

Editorial Team — Wed, 28 May 2025 00:17:05 +0000

Gilead Sciences has acquired exclusive ownership of two arenavirus-based immunotherapy programs for hepatitis B (HBV) and human immunodeficiency virus (HIV) from Hookipa Pharma. The transaction is the result of a long-standing collaboration between the two companies.
Under the terms of the agreement, Gilead will pay a total of $10 million to take full control of the HB-400 and HB-500 programs. Of that amount, $3 million is due at the close of the deal, with the remaining $7 million to be paid in three stages as the transfer is completed. The deal was signed on May 21, as disclosed in a May 22 filing with the Securities and Exchange Commission.
The HB-400 candidate, a therapeutic vaccine for chronic HBV, entered a Phase 1a/1b clinical trial in 2023. The HB-500 candidate, designed for HIV, is currently in an early-stage trial. As part of the new agreement, Hookipa will be responsible for winding down the HB-500 study. This trial had aimed to enroll approximately 30 participants and was previously scheduled to conclude in November 2025, according to ClinicalTrials.gov.

Both HB-400 and HB-500 utilize arenaviral backbones—specifically, lymphocytic choriomeningitis virus and pichinde virus—to deliver antigens targeting HBV and HIV, respectively.
The programs stem from a 2022 agreement between Gilead and Hookipa. At that time, Gilead made an upfront payment of $15 million and invested an additional $5 million in Hookipa equity. The 2022 pact included provisions for a $10 million program completion fee, up to $162.5 million in development milestones, and as much as $65 million in commercialization payments.
Gilead’s collaboration with Hookipa began in 2018 when it first licensed arenaviral immunization technologies from the biotech for use against HBV and HIV. In 2021, Gilead initially chose not to take an HIV candidate into the clinic. The company later revised its position, leading to the 2022 agreement that produced the HB-400 and HB-500 programs.
In addition to its development-stage assets, Gilead currently markets the antiretroviral drugs Biktarvy and Descovy, which together contributed to a combined $19.6 billion in HIV-related sales last year. The company also markets Vemlidy for HBV.
The HB-400 candidate represents a potential new offering in HBV, following recent announcements by other companies in the space. Vir Biotechnology and GSK have both discontinued efforts to develop “functional cures” for HBV this year, although GSK continues to work on an alternative candidate.

Hookipa Pharma, based in New York, has experienced recent business setbacks. These include the cancellation of a proposed merger with Poolbeg Pharma in February and workforce reductions following the termination of a collaboration with Roche on its HB-700 program for KRAS-mutated cancers.
Gilead’s acquisition of the HB-400 and HB-500 programs concludes its arenavirus collaboration with Hookipa and grants it full rights to the two therapeutic vaccine candidates.

Sanofi Acquires Vigil Neuroscience for $470M to Boost Alzheimer’s Drug Pipeline

Editorial Team — Wed, 28 May 2025 00:13:01 +0000

Sanofi is acquiring Vigil Neuroscience for $470 million to expand its Alzheimer’s drug portfolio, focusing on TREM2-targeting therapies like VG-3927. Learn how this acquisition strengthens Sanofi’s neurology and immunology strategy.
Sanofi is acquiring biotechnology company Vigil Neuroscience in a $470 million deal to strengthen its presence in Alzheimer’s disease treatment. The deal includes VG-3927, Vigil’s most advanced experimental drug, which targets the TREM2 protein associated with Alzheimer’s.
Sanofi had previously invested $40 million in Vigil, which gives the pharmaceutical giant a strategic edge if Vigil’s TREM2-amplifying drugs move toward licensing. VG-3927 recently completed early-phase trials in both healthy volunteers and Alzheimer’s patients. Vigil’s CEO, Ivana Magovčević-Liebisch, noted that the buyout will help accelerate development of this potentially transformative treatment.
TREM2 is considered a promising target in Alzheimer’s research because it activates immune responses in the brain when harmful amyloid-beta plaques are present. Malfunctioning TREM2 can lead to inflammation, nerve damage, and cell death—key factors in Alzheimer’s progression. Sanofi’s head of R&D, Houman Ashrafian, emphasized that TREM2 connects immune system dysfunction to neurological diseases, aligning with Sanofi’s expertise in immunology.

Although Sanofi will not acquire VGL101—another compound from the Vigil platform—those rights will revert to its original licensee, Amgen.
Sanofi has traditionally focused on immunology, oncology, and rare diseases, with neurology playing a relatively minor role. Its only marketed neurology drug, Aubagio for multiple sclerosis, accounted for less than 1% of biopharma sales last year. However, the company is actively expanding in this area. Several advanced-stage candidates—including frexalimab for MS, lupus, and type 1 diabetes, riliprubart for chronic inflammatory demyelinating polyneuropathy (CIDP), and tolebrutinib for MS—are in the pipeline. Tolebrutinib, acquired through a $3.7 billion deal with Principia Biopharma in 2020, is currently under FDA review with a decision expected by September.
The acquisition of Vigil supports Sanofi’s long-term strategy to address critical unmet needs in neurology through immunology-based therapies. Provided a majority of Vigil shareholders approve, the deal is expected to close between July and September. Key investors, including Atlas Venture and Bruce Booth—who collectively own 16.2% of Vigil shares—have already agreed to support the transaction.
In addition to the $8 per share offer, Vigil shareholders will receive a contingent value right worth $2 per share if VG-3927 achieves its first commercial sale. While Vigil’s stock opened at $14 when it went public in January 2022, its value has since dropped, making this acquisition a compelling exit. Analyst Myles Minter described the outcome as favorable for shareholders, given current market conditions and drug development progress.

This move reinforces Sanofi’s commitment to building a stronger foothold in Alzheimer’s research and broadening its neuroscience portfolio with immune-focused treatments.

Moderna Delays Flu-COVID Combo Vaccine Filing Amid FDA Request

Editorial Team — Thu, 22 May 2025 21:29:08 +0000

Moderna has announced that it is withdrawing its application for approval from the U.S. Food and Drug Administration (FDA) for its combination flu and COVID-19 vaccine, identified as mRNA-1083.
According to the company, it intends to submit the application again later in the year. The decision to resubmit will be timed to follow the availability of efficacy data from a phase 3 clinical trial of Moderna’s standalone flu vaccine, mRNA-1010.
The original application for mRNA-1083 had been filed last year. Based on that submission, the company had been on track to potentially secure FDA approval for the vaccine for adults aged 50 and above by November 2025. However, at the beginning of May, Moderna revised its expectations, shifting the anticipated timeline for approval to 2026. This adjustment followed a request from the FDA, in which the agency asked to review phase 3 efficacy data prior to making any decision on whether to authorize the vaccine.
The company has stated that the decision to withdraw the application was made voluntarily. Moderna now plans to refile the application once interim data from its ongoing phase 3 trial of mRNA-1010 are available. The trial is currently in progress, and the company expects to have preliminary results this summer.

Stephen Hoge, M.D., who serves as the president of Moderna, addressed the FDA’s request and the company’s response during an earnings call held at the beginning of May. He noted that Moderna is approaching the point at which data will become available from a 40,000-participant study evaluating the mRNA-1010 flu vaccine. In that context, Hoge remarked, “It makes good scientific sense that that would be a part of the review that’s going on for our flu-COVID combination.”
At the time of the call, Moderna had not yet decided whether it would include the data in an amendment to the existing biologics license application (BLA) or submit a new application altogether. “It is totally appropriate to submit that data as an amendment to the BLA,” Hoge said. “It could also be, from a pragmatic perspective, it makes sense to update more broadly the BLA submission with it, which could result in a resubmission.” He added that Moderna would proceed with whichever option appears most practical and aligns with the direction provided by the FDA.

Hoge further explained that Moderna’s interactions with the FDA have remained consistent. Describing the situation, he said it had been “business as usual” with regard to communications, and he stated that the company continues to maintain “productive exchanges” with the agency concerning all of its current file reviews.
Although the company has not specified an exact date for when it intends to resubmit the application, it has confirmed that the filing will follow the release of the phase 3 efficacy data from mRNA-1010 later this year.

Scientists Identify New Target to Prevent Cancer from Spreading to Bones

Editorial Team — Thu, 22 May 2025 20:13:16 +0000

In a major breakthrough, researchers have uncovered a potential way to stop cancer from spreading to the bones—a painful and common complication in many cancer patients. Using CRISPR gene-editing technology, scientists identified a protein called ACBP that appears to play a key role in bone metastasis, potentially opening the door to more effective treatments.

The research, led by Dr. Li Ma, an oncologist at the University of Texas MD Anderson Cancer Center, was published in Science Translational Medicine.

How the Discovery Works

The team used a CRISPR-based screening method to explore which genes might drive cancer to spread to bones. They introduced 2,302 guide RNAs into cancer cells, each designed to increase the expression of a different gene. This process revealed that cancer cells with higher levels of the ACBP protein were significantly more likely to metastasize to the bones.

Further analysis of human cancer genomes confirmed this finding: patients with elevated ACBP levels were also more prone to bone metastases. In lab experiments, mice with higher levels of ACBP in their bloodstream saw cancer spread to their bones more often. However, when researchers treated these mice with two compounds—etomoxir and IKE—the cancer did not reach the bones.

Why This Matters

Bone metastasis is a major concern for cancer patients. It often causes chronic pain and increases the risk of fractures, making life significantly harder for those affected. While current treatments like bisphosphonates help manage symptoms, they don’t stop the spread of cancer to the bones.

This new research marks a step forward. The compounds tested—etomoxir, which disrupts fatty acid metabolism, and IKE, which triggers a type of cell death known as ferroptosis—were effective at reducing bone metastases in mice. By blocking the metabolic pathways cancer cells use to survive and spread, these compounds show promise for future therapies.

A Growing Focus on Bone Metastasis

The importance of this discovery is highlighted by recent news that Former President Joe Biden has aggressive prostate cancer that has metastasized to his bones. Although his medical team reports that the condition is under control, the case has brought renewed attention to the urgent need for better treatments targeting bone metastasis.

This research not only sheds light on the molecular mechanisms behind bone metastasis but also provides a strong foundation for developing targeted therapies that could dramatically improve outcomes for cancer patients in the future.

CellCentric Secures $120M Series C Funding to Advance Myeloma Drug Inobrodib

Editorial Team — Thu, 22 May 2025 20:11:03 +0000

CellCentric, a UK-based biotechnology company backed by Pfizer, has raised $120 million in Series C funding to accelerate the development of its first-in-class multiple myeloma treatment, inobrodib. The funding will support pivotal clinical trials and advance the company’s drug development pipeline for relapsed or refractory multiple myeloma.

Currently in a Phase 2a clinical trial, inobrodib—an oral p300/CBP inhibitor—is being evaluated in combination with dexamethasone and Bristol Myers Squibb’s Imnovid (pomalidomide). The latest readout from the American Society of Hematology (ASH) 2024 Annual Meeting showed a promising 75% overall response rate at the highest treatment level.

Funding to Support Accelerated FDA Pathway

The newly raised funds will enable a Phase 2/3 trial targeting patients with heavily pre-treated multiple myeloma, with the goal of generating data for a potential accelerated approval application to the U.S. Food and Drug Administration (FDA).

CellCentric also plans to use part of the funding to:

Launch a Phase 3 clinical program in mid-2026
Initiate new studies of inobrodib in maintenance therapy settings, including combinations with bispecific antibodies

Strategic Investors Fueling Growth

The Series C round was co-led by RA Capital Management and Forbion, with additional participation from:

Pfizer
BrightEdge, the venture capital arm of the American Cancer Society
Avego Bioscience Capital

This latest raise builds on earlier investments, including a $25 million strategic investment from Pfizer in 2023 and $35 million from RA Capital in 2022.

Inobrodib works by targeting p300/CBP, key regulators of gene expression, to reduce the activity of oncogenic drivers MYC and IRF4—both known to fuel cancer progression in multiple myeloma and other malignancies.

CellCentric, originally spun out of research led by Dr. Azim Surani at the University of Cambridge, is actively expanding its U.S. presence. In April, the company opened a new office in Burlington, near Boston, to enhance its drug development capabilities and strengthen collaboration with North American partners.

Boehringer’s Nerandomilast Advances in IPF Amid Analyst Reservations

Editorial Team — Thu, 22 May 2025 20:06:32 +0000

Boehringer Ingelheim’s presentation of phase 3 data for its idiopathic pulmonary fibrosis (IPF) drug candidate nerandomilast has received a reserved response from analysts, who described the treatment as “a step forward but not game-changing.”
The company had previously disclosed in September that the study met its primary endpoint, raising expectations for a potential improvement over existing antifibrotic therapies, namely Boehringer’s Ofev and Roche’s Esbriet.

The study enrolled 915 patients, 78% of whom were receiving background antifibrotic treatment—535 on Ofev and 380 on Esbriet. Results showed that at Week 52, patients receiving a high dose of nerandomilast had a mean lung capacity decline of −114.7 ml, compared to −183.5 ml in the placebo group. Among those using Ofev, the decline was −118.5 ml with nerandomilast versus −191.6 ml on placebo. The smallest difference was observed in the subgroup of patients taking Esbriet, where the low dose of nerandomilast performed worse than placebo.
In a note dated May 19, Leerink Partners analysts commented that nerandomilast “looks to us to be an incrementally better Ofev,” but emphasized it remains a disease-slowing rather than disease-halting agent. They characterized it as “a decent Ofev-successor” but noted that its “modest effect size” was unlikely to prompt immediate changes in IPF treatment practices.
Concerns were also raised about the drug’s interactions with current therapies. According to Boehringer, drug-drug interactions between nerandomilast and Esbriet were responsible for the low dose’s failure to improve outcomes in that subgroup. In addition, overlapping toxicity was observed with Ofev. Diarrhea was the most frequently reported adverse event, affecting 41% of patients on the high dose of nerandomilast and occurring most often among those concurrently using Ofev.

Leerink analysts pointed out that this overlap in tolerability could limit the feasibility of using nerandomilast alongside Ofev. “Diarrhea leading to discontinuation was mainly observed with background [Ofev] use, which we think may limit the number of patients who are able to tolerate concurrent use,” they wrote.
While the analysts acknowledged that nerandomilast demonstrated a “solid profile that warrants approval,” they also highlighted the opportunity this leaves for other developers in the IPF space. The perceived limitations of nerandomilast were cited as a positive development for PureTech Health and MannKind, both of which are currently advancing their own IPF programs.

FDA Sets Tougher Approval Criteria for Covid Booster Shots in Healthy Individuals

Editorial Team — Thu, 22 May 2025 19:57:51 +0000

The Food and Drug Administration (FDA) has introduced more rigorous regulatory requirements for approving future COVID-19 booster shots, particularly for healthy individuals. This marks a significant shift from the previous approach, which typically approved annual boosters for all Americans based mainly on lab tests showing a strong immune response. Under the new guidance, pharmaceutical companies must now conduct clinical trials to prove that updated boosters are both safe and effective in healthy adults and children. This change could reduce the number of people eligible for routine boosters and increase costs for vaccine manufacturers.
Dr. Vinay Prasad, a vocal critic of pharmaceutical industry practices and now head of the FDA’s vaccine division, expressed skepticism about the blanket recommendation for repeated boosters in healthy individuals. He emphasized the lack of clear evidence supporting multiple annual doses for low-risk Americans and advocated for a more targeted approach.

In a paper published in the New England Journal of Medicine, FDA Commissioner Marty Makary and Dr. Prasad outlined a new risk-based strategy. For high-risk groups—including people over 65 and those with certain medical conditions such as obesity or depression—the FDA will continue to accept immunogenicity data as sufficient for approval. These individuals, estimated to number between 100 to 200 million, would still have relatively easy access to new vaccines.
However, for healthy individuals aged six months to 64 years without underlying health issues, the FDA will now require evidence from randomized, placebo-controlled trials. These trials must demonstrate that boosters reduce symptomatic COVID-19 cases by at least 30%, and participants must be monitored for at least six months to assess lasting protection. The new approach reflects the agency’s view that annual updates for all Americans are no longer scientifically justified, especially as the virus is now mutating more slowly than influenza.
The FDA stated that while it will continue to support rapid access for vulnerable populations, it expects drugmakers to conduct more thorough post-market studies in healthy groups. This approach seeks to strike a balance between ensuring safety and avoiding unnecessary vaccinations.

Financial analysts responded cautiously to the news. While acknowledging potential impacts on revenue for vaccine makers like Pfizer, BioNTech, and Moderna, they described the FDA’s stance as reasonable and science-based. Some noted that the new risk-based model aligns with international practices, where most high-income countries limit COVID booster recommendations to older or high-risk individuals.
The FDA’s paper also criticized the previous “one-size-fits-all” strategy, which it said may have eroded public trust in vaccination. Citing CDC data, it noted declining booster uptake, especially among children and even healthcare workers. The authors argued that unnecessary annual COVID shots could contribute to broader vaccine hesitancy, including for well-established vaccines like MMR (measles, mumps, rubella), which remain vital.Ultimately, the FDA’s revised guidance represents a shift toward a more evidence-driven, personalized approach to COVID-19 vaccination, with future approvals grounded in stronger data and differentiated by patient risk.

FDA Delays Biohaven’s Approval Decision for Rare Disease Drug Troriluzole

Editorial Team — Tue, 20 May 2025 23:17:38 +0000

The U.S. Food and Drug Administration (FDA) has postponed its decision on Biohaven Pharmaceuticals’ lead drug candidate, troriluzole, a potential treatment for a group of rare neurological conditions that damage nerve function. The company had anticipated a regulatory verdict by the end of September, following the completion of late-stage clinical trials. However, Biohaven announced that the FDA now requires additional time and input from an independent advisory committee, a move that adds uncertainty to the review process.

This development contradicts Biohaven’s previous communication. In its recent earnings report, the company stated that FDA officials had not mentioned the possibility of convening an advisory panel—a step usually taken when the agency has remaining questions about the safety, efficacy, or overall benefit-risk profile of a drug under review.

Troriluzole is Biohaven’s most advanced clinical candidate and is designed to metabolize into a molecule already approved in the U.S. and Europe to treat amyotrophic lateral sclerosis (ALS). Biohaven is positioning the drug as a therapy for a wider range of conditions, from the rare spinocerebellar ataxia (SCA) to more common illnesses like obsessive-compulsive disorder (OCD).

Key Developments and Market Impact

Regulatory setback in the U.S.: The FDA’s request for an advisory committee adds months to the review process and suggests increased regulatory scrutiny.
Unexpected reversal: Just days before the announcement, Biohaven reported no signs from the FDA of needing such a meeting.
European withdrawal: In March, Biohaven withdrew its marketing application for troriluzole in Europe after the EMA indicated likely rejection and declined to grant a special designation with economic benefits.
Market reaction: The European decision alone led to a $400 million loss in Biohaven’s market value. Combined with other setbacks, the stock is down roughly 15% over the past six months.
Investor concerns: Analysts such as Leonid Timashev of RBC Capital Markets say the new delay raises doubts about the overall regulatory outlook for troriluzole and increases the chances of a rejection.
Staffing issues at the FDA: Some believe the delay may also be partly attributed to internal staffing shortages at the FDA following recent layoffs.

If successful, troriluzole would become the first approved treatment for SCA and could mark a turning point for Biohaven, which has not brought a new therapy to market since it sold its migraine drug portfolio to Pfizer for $12 billion in 2022. The company has indicated it plans to reapply in Europe and remains committed to advancing its rare disease pipeline.

Pathos AI Secures $365 Million in Series D Funding to Accelerate AI-Powered Cancer Drug Development

Editorial Team — Tue, 20 May 2025 22:47:06 +0000

Pathos AI, a clinical-stage biotech company leveraging artificial intelligence to revolutionize cancer treatment, has announced the successful close of a $365 million Series D funding round. The funding will support the development of AI-driven oncology therapeutics, including solid tumor drug candidates from Novo Nordisk and Prelude Therapeutics.

The latest investment round values Pathos at an estimated $1.6 billion, reflecting strong investor confidence in the company’s proprietary PathOS Platform, which integrates multimodal data—including molecular, clinical, and imaging datasets—to optimize oncology drug development.

Focus on Clinical-Stage Pipeline: Pocenbrodib and P-500

A key focus of the Series D funding will be the continued advancement of pocenbrodib, a CBP/p300 inhibitor licensed from Novo Nordisk in 2023. Originally developed by Forma Therapeutics, pocenbrodib is currently undergoing Phase 1b/2a clinical trials for metastatic castration-resistant prostate cancer (mCRPC). The trials are evaluating the drug both as a monotherapy and in combination with Zytiga, Lynparza, and Pluvicto.

Pathos believes pocenbrodib holds promise beyond prostate cancer, with potential applications across a broad range of solid tumor types.

In addition, Pathos plans to initiate clinical development of P-500, a brain-penetrant PRMT5 inhibitor licensed from Prelude Therapeutics, later in 2024. Prelude previously evaluated P-500 in a Phase 1 trial involving patients with uveal melanoma and high-grade gliomas.

Leveraging AI to Revolutionize Oncology

Pathos’ AI-powered PathOS Platform is at the core of its strategy, enabling the identification of predictive biomarkers and the design of precision-targeted clinical trials. The company aims to use the new capital to accelerate platform enhancements, improve patient stratification, and increase the speed and success rate of oncology drug development.

“Our mission at Pathos is to transform oncology drug development by harnessing the full potential of multimodal data and artificial intelligence,” said Iker Huerga, the newly appointed CEO of Pathos AI. Huerga previously served as Chief Data Scientist for Oncology R&D at AstraZeneca.

Continued Growth and Strategic Acquisitions

The Series D round included participation from both existing backers and new investors. This follows a $62 million Series C round led by New Enterprise Associates (NEA) just seven months ago.

In a strategic move last December, Pathos acquired Rain Oncology, adding the Phase 3-stage MDM2 inhibitor milademetan to its pipeline. However, the company has yet to comment on future plans for the asset, which previously failed in liposarcoma trials.

About Pathos AI

Pathos AI is a Boston-based biotechnology company applying advanced artificial intelligence and multimodal data analytics to accelerate the discovery and development of next-generation oncology therapeutics. Through its proprietary PathOS Platform, the company is redefining how cancer treatments are identified, developed, and delivered.

Life Sciences Voice Top Five Newsletter

Editorial Team — Tue, 20 May 2025 22:36:59 +0000

Welcome to this week’s edition of the Life Sciences Voice Top Five Newsletter, your go-to source for the latest breakthroughs in the life sciences industry! This time, we’re looking at Eli Lilly’s $250 million investment in Purdue University, FDA’s decision to halt chikungunya vaccine in adults, the first-ever FDA-approved at-home cervical cancer screening kit, and more. Stay informed and inspired by the innovations driving life sciences forward!

Eli Lilly Expands Purdue Partnership with $250 Million Investment

Eli Lilly will invest up to $250 million through 2032 to expand its partnership with Purdue University. The collaboration focuses on AI-driven drug discovery, clinical trials, regulatory and manufacturing efficiency, and workforce development in Indiana’s life sciences sector. Purdue researchers will work on campus in West Lafayette and with Lilly scientists in Indianapolis and Lebanon. The partnership builds on previous agreements totaling over $100 million since 2017 and includes ongoing initiatives like the Lilly Scholars program and the Young Institute Pharmaceutical Manufacturing Consortium, which targets advancements in drug production technologies.

U.S. Recommends Suspension of Ixchiq Vaccine Use in Older Adults Amid Safety Review

The FDA and CDC have advised pausing the use of Valneva’s chikungunya vaccine, Ixchiq, in individuals aged 60 and older while investigations into serious adverse events (SAEs) continue. This follows similar actions by France and the European Medicines Agency, which cited 17 SAEs, including two deaths, in vaccine recipients aged 62 to 89. The CDC previously recommended caution for those 65 and older. Valneva, which has distributed over 40,000 doses globally, stated that most reported events involved older adults with pre-existing conditions and confirmed ongoing coordination with global health authorities.

FDA Approves First At-Home HPV Self-Collection Kit for Cervical Cancer Screening

The U.S. FDA has approved the Teal Wand, the first at-home prescription kit for HPV screening and cervical cancer risk assessment. Developed by Teal Health, the device uses a spongelike swab for self-collection without a speculum. Samples are mailed to a lab and tested using standard Pap smear methods. A study of over 600 participants found accuracy comparable to clinician-collected samples, with 94% preferring at-home screening. The device is for women aged 25 to 65 at average risk and will launch in California via Teal Health’s telehealth platform in June, with national expansion to follow.

Abeona Therapeutics Sells Priority Review Voucher for $155 Million After FDA Approval of Zevaskyn

Abeona Therapeutics sold a Priority Review Voucher (PRV) for $155 million shortly after receiving FDA approval for Zevaskyn, its gene therapy for recessive dystrophic epidermolysis bullosa (RDEB). The buyer was not disclosed. Zevaskyn, a one-time autologous cell-based therapy, is applied as a sheet to wounds to promote healing and reduce pain. Abeona plans a phased commercial launch in Q3 2025, initially treating 10 to 15 patients. Proceeds from the PRV sale are expected to fund operations for over two years. The transaction comes amid regulatory uncertainty and limited capital-raising options due to a depressed stock price.

Johnson & Johnson’s Gene Therapy for XLRP Falls Short in Phase 3 Trial

Johnson & Johnson’s gene therapy botaretigene sparoparvovec (bota-vec) did not meet its primary goal of improving vision-guided mobility in a phase 3 trial for X-linked retinitis pigmentosa (XLRP), a rare genetic eye disease. The LUMEOS study involved 95 participants, with 58 receiving a single injection of bota-vec. While secondary endpoints, such as patient-reported vision and letter chart assessments, showed improvement, the results lacked statistical significance. All treated participants experienced adverse events, with 53% linked to the therapy. Johnson & Johnson, which acquired full rights to bota-vec in a $415 million deal in December 2023, is evaluating its next steps.

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