Mines D`or Orbec Inc

bluebird bio, Inc. (NASDAQ:BLUE) ranks 9th among promising gene editing stocks, with 18 hedge funds holding shares worth $16.49 million. Despite safety concerns and Nasdaq non-compliance, BLUE's Q2 2024 revenue surged to $16.1 million, and it has FDA-approved therapies for severe genetic disorders. The global genome editing market is expected to grow to $38.19 billion by 2033, driven by increasing prevalence of genetic conditions and advancements in CAR-T cell therapies.

Pfizer's sickle cell disease therapy Oxbryta was pulled from market due to risks, highlighting the challenges of accelerated approval. The FDA's program has brought nearly 300 drugs to market, often years earlier, but success rates vary. While many drugs have secured traditional approval, others like Oxbryta and Aduhelm were ultimately unsuccessful. The pathway relies on biomarkers predicting clinical benefit, but confirmatory trials are often delayed, raising concerns about safety and efficacy. Experts argue for tighter deadlines and better validation of biomarkers to improve the program's reliability.

Kendric Cromer, the first patient to undergo recently approved gene therapy for sickle cell disease, has left the hospital after 44 days of treatment. The U.S. FDA approved two gene therapies in December 2023, offering a potential cure for the disease. Cromer and his family faced significant emotional and physical challenges during the treatment.

Nanoscope Therapeutics plans to file a BLA for MCO-010 to treat retinitis pigmentosa. Seven patients treated with bluebird bio's eli-cel developed hematologic malignancies. Poseida Therapeutics' P-BCMA-ALLO1 showed a 91% ORR in r/r multiple myeloma. Kiromic BioPharma's KB-GDT-01 provided disease stabilization in NSCLC. Adicet Bio's ADI-100 received FDA clearance for additional patient recruitment in lupus nephritis trial. ElevateBio's LNP platform successfully edited liver genes in mice with low immunogenicity.

CGTs disrupt treatment for genetic diseases, with 69 marketed worldwide and 7 novel FDA approvals in 2023. Oncology leads CGTs, projected to reach $37bn by 2030. Outsourcing vs. in-house manufacturing is crucial, with Lonza Group as the leading CMO for marketed CGTs.

AstraZeneca to pay $2B to CSPC for a cardiovascular drug, Astellas Pharma licenses gene therapy from AviadoBio, Merck partners with Mestag Therapeutics for fibrosis treatments, Insitro signs three agreements with Eli Lilly for metabolic diseases, MiNK Therapeutics and Autonomous Therapeutics collaborate on metastatic tumors, Bayer and MOMA Therapeutics partner in oncology, Wonderskin names 5W Public Relations as AOR, Eversana signs with Oracle Argus Cloud, Qinotto and Eli Lilly collaborate, Onspire Health acquires Aesthetic Brand Marketing, Reunion Neuroscience partners with Seleni Institute, Danco Laboratories to seek FDA approval for Mifeprex use in miscarriages, Exact Sciences' Cologuard Plus wins FDA approval, Boehringer Ingelheim's survodutide gets Breakthrough Therapy designation, Lunds & Byerlys recalls Lone Star Dip, Kezar Life Sciences' zetomipzomib development halted by FDA, Bayer's elinzanetant NDA accepted by FDA, Biogen's felzartamab gets Breakthrough Therapy Designation, Black Diamond Therapeutics to cut jobs, Upstream Bio targets $182M for IPO, City Therapeutics launches with $135M, Purespring Therapeutics raises $105M, Judo Bio launches with $100M, Orasis Pharmaceuticals raises $78M, CAMP4 Therapeutics targets $67M for IPO, Basecamp Research closes $60M Series B, Arda Therapeutics raises $43M Series A, Crinetics Pharmaceuticals announces public offering, GSK to pay $2.2B to settle Zantac lawsuits, Scholar Rock's SMA drug shows muscle function improvement, CDC to screen travelers due to Marburg virus outbreak in Rwanda, Ad Council, AMA, and CDC launch flu vaccination campaign, Sage Therapeutics' Sage-718 fails in Alzheimer's study, WeightWatchers adds GLP-1 obesity treatments, Gritstone Bio files for Chapter 11, BioPharmCatalyst launches AI-powered chatbot Aiden, Otsuka Precision Health and Amalgam Rx launch Alzheimer's app Elevmi, PatientPoint unveils interactive communication solution, Curia rebrands, Switzerland's COMCO terminates Novartis investigation, Bluebird Bio's Skysona linked to blood cancer in seven children, Solera Health rebrands, HearingLife launches pickleball sweepstakes, Alfasigma launches Morpho brand, Healthy Minds Innovations launches Our World Of Emotions campaign.

New data shows seven children who took part in bluebird bio's gene therapy Skysona clinical studies developed haematologic cancers. Researchers found one patient with haematological cancer from ALD-102 and six from ALD-104, with six cases of myelodysplastic syndrome and one of acute myeloid leukaemia. The risk of secondary malignancies is a known concern with gene therapies using lentiviral vectors.

Regeneron, despite being a latecomer in cancer therapies, aims to leverage its packed pipeline to become a leader in the next wave of oncology treatments. Dr. Israel Lowy, senior vice president of translational and clinical sciences in oncology, highlights the company's focus on combinations like Libtayo and fianlimab, which could rival existing treatments like Opdualag and Keytruda. Regeneron's journey includes pioneering bispecific antibodies and facing early challenges, but with ongoing clinical trials and collaborations, the company seeks to establish itself as a major player in cancer therapy.

Seven children treated with bluebird bio's Skysona for cerebral adrenoleukodystrophy developed blood cancers, including six cases of myelodysplastic syndrome and one of acute myeloid leukemia, linked to clonal vector insertions and somatic mutations in cancer-related genes. Skysona's label warns of hematologic malignancies, and concerns about secondary blood cancers have been previously raised.

The 2024 Cell & Gene Meeting on the Mesa focused on overcoming challenges in scaling production and delivery of cell and gene therapies, emphasizing the importance of learning from failures and regulatory convergence to accelerate patient access to new technologies.