NEW YORK – Mount Sinai Health System said last week that it has opened the Hamilton and Amabel James Center for Artificial Intelligence and Human Health. The center, which a spokesperson said took five years to complete at a cost of about $100 million, will combine artificial intelligence with data science and genomics. It is located at the Mount Sinai Hospital campus in Manhattan and will initially house approximately 40 principal investigators, as well as 250 graduate students, postdoctoral fellows, computer scientists, and support staff. It was in part supported by a gift from Hamilton James, executive VP of investment firm Blackstone, and his wife Amabel James. The center will house several institutes, including the Institute for Genomic Health and Division of Medical Genetics and the Institute for Personalized Medicine.
Milan, Italy-based AAVantgarde Bio this week said the US Food and Drug Administration has granted it orphan drug designation for the company's lead program, AAVB-081. AAVB-081 is a gene therapy being developed as a treatment for retinitis pigmentosa caused by Usher syndrome type 1B, an inherited disease that affects the retina and the inner ear, and which can lead to progressive vision loss. The FDA grants orphan designation to drugs for rare diseases, which provides sponsors tax credits for clinical trials, exemption from user fees, and seven years of market exclusivity for the drug after regulatory approval.
Lantern Pharma this week said the US Food and Drug Administration granted fast-track designation to its investigational therapy for triple-negative breast cancer, LP-184. The company is evaluating the drug in a Phase Ia trial in advanced and metastatic solid tumors. Under fast-track designation, sponsors can meet with the FDA more frequently to get advice on developing fast-track designated agents, submit data on a rolling basis, and apply for accelerated approval or priority review.
Latern developed LP-184 using its proprietary RADR AI platform to identify patient populations that are likely to respond best to treatment. Analysis using the platform and in vivo studies showed that PTGR1 expression is often elevated in TNBC tumors compared to normal tissue, and these tumors were particularly responsive to LP-184. "This biomarker-driven approach allows for the potential identification of patients most likely to respond to LP-184 therapy, aligning with current and emerging precision medicine approaches in TNBC treatment," the company said in a statement.
The US Food and Drug Administration has granted orphan drug designation to EXG110, Exegenesis Bio's gene therapy candidate for Fabry disease, the Philadelphia-based company said this week. The first patient to receive this gene therapy has been dosed within a clinical trial in China, and Exegenesis said that it plans to initiate a clinical trial in the US next year.
Oxford, UK-based AlveoGene this week said the US Food and Drug Administration has granted orphan drug designation to AVG-002, a gene therapy candidate that the company is developing for neonatal surfactant protein B deficiency, an extremely rare genetic disorder that causes fatal respiratory distress syndrome in newborns. AlveoGene said it is preparing to advance the gene therapy, which patients would take by inhaling it, into clinical trials and could potentially file for marketing authorization by 2028.
Adaptimmune said this week that the first sarcoma patient has been treated commercially with the autologous cell therapy Tecelra (afamitresgene autoleucel) at the Moffitt Cancer Center. The US Food and Drug Administration approved Tecelra in August for patients whose tumors express the MAGE-A4 antigen and who have one of four human leukocyte antigen types: HLA A*02:01P, HLA A*02:02P, HLA*02:03P, or HLA*02:06P. The T-cell receptor (TCR) T-cell therapy's list price is $727,000 dollars per infusion.
The US Food and Drug Administration this week granted breakthrough therapy designation to Merck and Kelun-Biotech's antibody-drug conjugate sacituzumab tirumotecan (sac-TMT) in advanced non-small cell lung cancer patients whose tumors harbor EGFR exon 19 deletion or exon 21 L858R mutations. Under the terms of a collaboration agreement, Kelun-Biotech maintains the rights to develop and manufacture sac-TMT in China, and Merck has the rights to develop it elsewhere. Agents with breakthrough therapy designation are eligible for more intensive guidance from the FDA, a scientific liaison to help accelerate review time, and the potential for priority review, contingent on certain criteria.
Health management platform company Function Health said this week that it will make Grail’s Galleri test available to eligible members. The company offers more than 100 laboratory tests, along with healthcare advice, to its more than 100,000 members in the US for $499 per year. Grail’s multi-cancer early detection blood test screens for several types of cancer and is recommended for adults with an elevated risk of cancer, including those age 50 years or older.
Precision medicine firm OncoHost said this week that its Cary, North Carolina-based CLIA lab has received accreditation from the College of American Pathologists (CAP). The company runs its proteomic PROphet blood test out of the laboratory. PROphet measures the levels of roughly 7,000 proteins, analyzing expression patterns to predict whether a patient is likely to respond to a given cancer therapy. The company’s initial offering is intended to guide decision-making around first-line treatment with immunotherapy in individuals with non-small cell lung cancer.
Alpro Group, Malaysia’s largest prescription pharmacy chain, said this week that it has signed a memorandum of understanding with AGTC Genomics. Under the agreement, AGTC will provide pharmacogenomics and nutrigenomics testing services, which will be available at Alpro pharmacies across the country. The partners will also launch educational initiatives to raise awareness about genetic testing.
Tokyo-based Eisai and Cambridge, Massachusetts-based Biogen this week announced that Mexico's Federal Commission for the Protection Against Sanitary Risk has approved the companies' beta-amyloid-targeting drug Leqembi (lecanemab) as a treatment for early Alzheimer's disease. Leqembi is already approved in the US, Japan, China, South Korea, Hong Kong, Israel, the United Arab Emirates, and Great Britain.
UC Davis Health said this week that it has received a $500,000 grant from the California health insurer Health Net to improve patient access to CAR T-cell therapies and clinical trials. The grant helps UC Davis expand access to these cell therapies into and beyond 33 counties around the Bay Area at a lower cost to patients. It will also support the development of new CAR T-cell products at the UC Davis Stem Cell Program's Alpha Clinic.
Foundation Medicine has awarded two grants of undisclosed amounts to organizations working to address disparities in biomarker testing for breast and prostate cancer, the company announced this week. The company originally issued an open call for grant proposals in April, requesting that they include collaboration between two separate organizations. The 2024 grant recipients are: Living Beyond Breast Cancer and Sidney Kimmel Comprehensive Cancer Center at Jefferson Health for a project titled "No One Missed: Grassroots Pilot for Under-Resourced Metastatic Breast Cancer Patients;" and Patient Advocate Foundation and Zero Prostate Cancer for a project titled "A Patient-Focused Partnership to Advance Health Equity in Biomarker Testing Among Patients with Prostate Cancer."
In Brief This Week is a selection of news items that may be of interest to our readers but had not previously appeared on Precision Medicine Online.