Orchard Therapeutics plc (ORTX)

Completion of enrollment achieved nearly one year ahead of schedule Primary analysis anticipated two years post-treatment TOKYO and LONDON and BOSTON, July 07, 2025 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a Kyowa Kirin company, today announced the last patient has been treated in a registrational trial evaluating the efficacy and safety of OTL-203, an investigational hematopoietic stem cell (HSC) gene therapy, in patients with the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). The trial (NCT06149403), referred to as HURCULES, compares treatment with OTL-203 to standard of care with allogeneic hematopoietic stem cell transplant (allo-HSCT).

LONDON, MADRID, and BOSTON, Feb. 28, 2025 (GLOBE NEWSWIRE) --  Orchard Therapeutics, a Kyowa Kirin company, announced it has recently reached an agreement with the Interministerial Commission for the Pricing of Medicines of the Directorate of Common Portfolio of Services of the Spanish National Health System (SNS) which will result in reimbursed access to Libmeldy® (atidarsagene autotemcel) for all eligible children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ) or early symptomatic early juvenile (ESEJ)—collectively referred to as early-onset—metachromatic leukodystrophy (MLD).

First U.S. patients being treated with Lenmeldy™ (atidarsagene autotemcel) in a commercial setting; other launch efforts progressing well following Food and Drug Administration approval last year

Featured data showcase the transformative potential of HSC gene therapy to enable cross-correction and restore enzymatic function in a variety of tissues and organs, including the CNS Featured data showcase the transformative potential of HSC gene therapy to enable cross-correction and restore enzymatic function in a variety of tissues and organs, including the CNS

Ten presentations at ESGCT 2024 detail the therapeutic potential and applicability of the company's platform in rare neurometabolic diseases and beyond

ISTANBUL and LONDON, and BOSTON, Oct. 07, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe and Er-Kim, an international pharmaceutical company specializing in the commercialization of novel therapies, today announced the companies have signed an exclusive distribution agreement to commercialize Libmeldy® (atidarsagene autotemcel), the only approved therapy for eligible children with early-onset metachromatic leukodystrophy (MLD).

LONDON and BOSTON, Aug. 28, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced nine presentations from across its late-stage neurometabolic hematopoietic stem cell (HSC) gene therapy portfolio will be featured at the Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium taking place September 3-6 in Porto, Portugal.

A nomination to add MLD to the U.S. Recommended Uniform Screening Panel has been submitted by a multi-disciplinary expert working group

Sorrento Therapeutics (OTCMKTS:SRNEQ – Get Free Report) and bluebird bio (NASDAQ:BLUE – Get Free Report) are both small-cap medical companies, but which is the better stock? We will compare the two businesses based on the strength of their earnings, analyst recommendations, dividends, valuation, risk, profitability and institutional ownership. Profitability This table compares Sorrento Therapeutics and bluebird bio’s net margins, return on equity and return on assets. Net Margins Return on Equity Return on Assets Sorrento Therapeutics N/A N/A N/A bluebird bio -419.64% -34.32% -14.44% Risk and Volatility Sorrento Therapeutics has a beta of 2.25, suggesting that its share price is 125% more volatile than the S&P 500. Comparatively, bluebird bio has a beta of 0.82, suggesting that its share price is 18% less volatile than the S&P 500. Analyst Ratings This is a breakdown of recent ratings and recommmendations for Sorrento Therapeutics and bluebird bio, as provided by MarketBeat.com. Sell Ratings Hold Ratings Buy Ratings Strong Buy Ratings Rating Score Sorrento Therapeutics 0 0 0 0 N/A bluebird bio 1 6 4 0 2.27 bluebird bio has a consensus price target of $5.74, indicating a potential upside of 457.71%. Given bluebird bio’s higher possible upside, analysts plainly believe bluebird bio is more favorable than Sorrento Therapeutics. Institutional and Insider Ownership 0.0% of Sorrento Therapeutics shares are held by institutional investors. Comparatively, 87.4% of bluebird bio shares are held by institutional investors. 2.6% of Sorrento Therapeutics shares are held by company insiders. Comparatively, 2.1% of bluebird bio shares are held by company insiders. Strong institutional ownership is an indication that hedge funds, endowments and large money managers believe a stock will outperform the market over the long term. Earnings & Valuation This table compares Sorrento Therapeutics and bluebird bio’s gross revenue, earnings per share and valuation. Gross Revenue Price/Sales Ratio Net Income Earnings Per Share Price/Earnings Ratio Sorrento Therapeutics $62.84 million 0.26 -$572.84 million N/A N/A bluebird bio $21.73 million 5.18 -$266.58 million ($0.74) -1.39 bluebird bio has lower revenue, but higher earnings than Sorrento Therapeutics. Summary Sorrento Therapeutics beats bluebird bio on 6 of the 11 factors compared between the two stocks. About Sorrento Therapeutics (Get Free Report) Sorrento Therapeutics, Inc., a clinical and commercial stage biopharmaceutical company, develops a portfolio of next-generation treatments for three therapeutic areas: cancer, infectious disease,and pain. It operates through two segments, Sorrento Therapeutics and Scilex. The company provides cancer therapeutic by leveraging its proprietary G-MAB antibody library and targeted delivery modalities, which include chimeric antigen receptor T-cell therapy (CAR-T), dimeric antigen receptor T-cell therapy, and antibody drug conjugate, as well as bispecific antibody approach; and Sofusa, a drug delivery technology that deliver biologic directly into the lymphatic system. Its clinical programs in development include anti-CD38 CAR-T therapy for the treatment of multiple myeloma, as well as for amyloidosis and graft versus host disease. The company develops resiniferatoxin, a non-opioid-based TRPV1 agonist neurotoxin for late stage cancer and osteoarthritis pain treatment; and ZTlido, a lidocaine topical system for the treatment of post-herpetic neuralgia. It engages in the development of SEMDEXA, an injectable viscous gel formulation, which is Phase III trial for the treatment of sciatica, a pathology of low back pain; SP-103, an investigational non-aqueous lidocaine topical system undergoing clinical development in chronic low back pain condition; and SP-104, a novel low-dose delayed-release naltrexone hydrochloride formulation for the treatment of fibromyalgia. It has collaboration with SmartPharm Therapeutics Inc. to develop gene-encoded antibody vaccine to protect against COVID-19; and Mayo Clinic for Phase Ib pilot study using sofusa lymphatic drug delivery technology to deliver Ipilimumab in patient with melanoma. The company was founded in 2006 and is based in San Diego, California. On February 13, 2023, Sorrento Therapeutics, Inc., along with its affiliate, filed a voluntary petition for reorganization under Chapter 11 in the U.S. Bankruptcy Court for the Southern District of Texas. The plan was later approved as Chapter 11 liquidation on November 30, 2023. About bluebird bio (Get Free Report) bluebird bio, Inc., a biotechnology company, researches, develops, and commercializes gene therapies for severe genetic diseases. Its product candidates for severe genetic diseases include ZYNTEGLO (betibeglogene autotemcel) for the treatment of transfusion-dependent ß-thalassemia; lovotibeglogene autotemcel for the treatment of sickle cell disease (SCD); and SKYSONA (elivaldogene autotemcel) to treat cerebral adrenoleukodystrophy. The company's clinical development programs include HGB-205, HGB-206, and HGB-210 to evaluate the safety and efficacy of lovo-cel in the treatment of patients with SCD; and HGB-204, HGB-205, HGB-207, and HGB-212 to evaluate the safety and efficacy of beti-cel in the treatment of patients with ß-thalassemia. It has license agreement with Orchard Therapeutics Limited. The company was formerly known as Genetix Pharmaceuticals, Inc., and changed its name to bluebird bio, Inc. in September 2010. bluebird bio, Inc. was incorporated in 1992 and is headquartered in Somerville, Massachusetts.

Eight presentations showcase the broad applicability of HSC gene therapy to address rare neurometabolic diseases and beyond Eight presentations showcase the broad applicability of HSC gene therapy to address rare neurometabolic diseases and beyond

TOKYO, LONDON and BOSTON, May 03, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced that Bobby Gaspar, M.D., Ph.D., co-founder and chief executive officer, has been named to the inaugural 2024 TIME100 Health, an annual list recognizing the impact, innovation, and achievement of the world's most influential individuals in health this year. The full list and related tributes will appear in the May 13, 2024 issue of TIME and is available online at https://time.com/collection/time100-health/.

Five specialized treatment centers being qualified across the U.S. to minimize travel burden on eligible patients and their families

One-time treatment with Lenmeldy has shown the potential to restore enzymatic function to stop or slow disease progression, with up to 12 years of follow-up (median 6.76 years) One-time treatment with Lenmeldy has shown the potential to restore enzymatic function to stop or slow disease progression, with up to 12 years of follow-up (median 6.76 years)

The U.S. Food and Drug Administration said on Monday it had approved UK-based Orchard Therapeutics' gene therapy for the treatment of children with a rare hereditary disease called metachromatic leukodystrophy (MLD).

Gene therapy being developed as a one-time treatment to address the underlying cause of the disease by inserting a functional copy of the human IDUA gene into a patient's own hematopoietic stem cells

Three oral presentations and seven posters highlight transformative potential of HSC gene therapy and newborn screening to address severe neurometabolic diseases Three oral presentations and seven posters highlight transformative potential of HSC gene therapy and newborn screening to address severe neurometabolic diseases