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Orchard Therapeutics plc (ORTX) is a pioneering biotechnology company dedicated to transforming the lives of children with life-threatening rare diseases through advanced gene therapies. The company focuses on the development, manufacturing, and commercialization of ground-breaking hematopoietic stem cell (HSC) gene therapies, which hold the potential to provide one-time treatments that address the underlying causes of severe genetic disorders.
Founded in 2015, Orchard Therapeutics collaborates with prestigious research centers globally to harness the life-saving potential of gene therapy. The company’s leadership team and partners bring over a decade of experience in advancing therapies for orphan diseases.
Orchard’s flagship product, Libmeldy® (atidarsagene autotemcel), is designed for the treatment of early-onset metachromatic leukodystrophy (MLD), a rare metabolic disorder. Libmeldy has received approval from the European Commission and the UK Medicines and Healthcare products Regulatory Agency and is under Priority Review by the U.S. FDA with a decision expected by March 18, 2024. The therapy aims to halt or slow disease progression by restoring enzymatic function.
The company is also advancing OTL-203 for mucopolysaccharidosis type I Hurler’s syndrome (MPS-IH) and OTL-201 for mucopolysaccharidosis type IIIA (MPS-IIIA), also known as Sanfilippo syndrome. These programs utilize the same HSC gene therapy platform as Libmeldy.
In a strategic move, Kyowa Kirin recently acquired Orchard Therapeutics, enhancing its portfolio with Orchard's clinically differentiated gene therapy platform. This acquisition is expected to drive the development of new treatments and support ongoing and future launches, including the continued growth of Libmeldy in Europe and potential new approvals in the U.S.
Orchard Therapeutics continues its mission to deliver curative gene therapies for severe diseases by translating promising early-stage research into approved treatments available to patients worldwide. The team remains committed to leveraging its extensive experience and cutting-edge technology to address unmet medical needs in rare and underserved conditions.
The "Europe Cell and Gene Therapy Market - Focused Insights 2023-2028" report, added by ResearchAndMarkets, provides a comprehensive analysis of the European market valued at $2.17 billion in 2022 and projected to reach $15.15 billion by 2028, marking a 38.20% CAGR. Key highlights include Zolgensma's revenue of $1.37 billion in 2022, with potential growth to $5.00 billion by 2026. The report examines over 1,564 clinical trials in Europe and profiles leading companies like Novartis, Gilead Sciences, and Legend Biotech. It emphasizes the importance of government and academic financing, contributing to market growth.
Technavio reports that the global gene therapy market is projected to grow by USD 4,613.9 million from 2022 to 2027, at a CAGR of 19.91%. This growth is fueled by increasing special drug designations such as orphan, fast track, and breakthrough designations by regulatory bodies, enhancing market opportunities. The oncology segment will see significant growth due to the unmet demand for cancer treatments. North America is expected to contribute 39% to the market growth, supported by government funding for gene therapy research. However, high treatment costs, ranging from USD 1 million to 3 million, present a major challenge.
The gene therapy market is projected to grow by $4.61 billion from 2023 to 2027, with a CAGR of 19.91%. This growth is attributed to increasing special drug designations, benefits of gene therapy, and rising chronic diseases prevalence.
The market report analyzes key areas including market size, trends, growth drivers, and challenges, covering around 25 vendors. The report segments the market by therapy areas like oncology, CNS, and rare diseases, along with delivery modes and geography.
Key vendors include Adaptimmune Therapeutics (ADAP) and Orchard Therapeutics (ORTX), among others.
Orchard Therapeutics (Nasdaq: ORTX) announced a change in its American Depositary Share (ADS) to ordinary share ratio, effective March 10, 2023. The new ratio is one ADS to ten ordinary shares, equivalent to a one-for-ten reverse ADS split. This adjustment aims to help the company comply with Nasdaq's minimum bid price requirement. Existing ADS holders will have their ADSs automatically exchanged, maintaining their percentage interest in the company's equity. This change does not affect Orchard's underlying ordinary shares, and fractional ADSs will be aggregated and sold, with proceeds distributed to ADS holders, as no fractional ADSs will be issued.
Orchard Therapeutics (ORTX) announced key business updates and financial results for 2022. The company reported revenue from Libmeldy of $18.8 million, with $5.8 million generated in Q4 2022. Following a productive FDA meeting, a pre-BLA meeting for OTL-200 is scheduled for Q2 2023. Strategic financing will provide up to $188 million to support regulatory submissions and operations. The company confirmed three MLD cases from newborn screenings, with ongoing commercial expansion in Europe. Cash, equivalents, and investments as of Dec 31, 2022, were $143.8 million, with anticipated declining cash burn in 2023 due to increasing revenues.
Orchard Therapeutics (ORTX) announced a securities purchase agreement for a private placement aimed at raising up to $188 million to enhance its gene therapy operations and extend its cash runway into 2025. The initial closing, expected by March 10, 2023, will yield $34 million from the sale of units at $6.00 each. Proceeds will be allocated to expand commercialization of Libmeldy, advance clinical programs, and service existing debt. The financing, led by RA Capital Management, reflects significant investor confidence ahead of the anticipated regulatory filing for OTL-200, targeting approval for treating metachromatic leukodystrophy (MLD).
BOSTON and LONDON, March 03, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX) announced management team presentations at two upcoming investor conferences. The TD Cowen 43rd Annual Healthcare Conference will take place on March 7, 2023, at 10:30 a.m. ET in Boston, focusing on orphan neurology drug discovery. The Oppenheimer 33rd Annual Healthcare Conference is scheduled for March 13, 2023, at 2:00 p.m. ET virtually. Live webcasts will be accessible on the company’s website, with replays archived afterward. Orchard is dedicated to advancing gene therapies aimed at severe genetic diseases, leveraging hematopoietic stem cell technology.
Orchard Therapeutics (Nasdaq: ORTX) has secured a reimbursement agreement with the New Therapies (NT) Council for its gene therapy, Libmeldy® (atidarsagene autotemcel), for metachromatic leukodystrophy (MLD) patients in Sweden. This follows similar agreements in the UK, Italy, and Germany, bolstering the company’s European expansion strategy. Libmeldy has shown impactful clinical results over 10 years of follow-up and aims to enhance access for patients across the Nordic region. The therapy is approved for early-onset MLD and is the first of its kind in the EU.
Orchard Therapeutics (Nasdaq: ORTX) presented long-term results from an updated analysis of the gene therapy OTL-200 for metachromatic leukodystrophy (MLD) at the 19th Annual WORLD Symposium™. The therapy demonstrated significant improvement in severe motor impairment-free survival, showing benefits with up to 11 years of follow-up. Among 39 patients treated, 17 of 18 pre-symptomatic late infantile patients maintained the ability to walk. No treatment-related serious adverse events occurred, and overall safety was favorable. The data aims to support potential U.S. approval for OTL-200, which is already approved in the EU. The findings underscore the promise of gene therapy in treating severe genetic diseases.
Orchard Therapeutics (Nasdaq: ORTX) announced a significant change in its American Depositary Shares (ADS) ratio, moving from 1:1 to 1:10, effective March 10, 2023. This reverse ADS split aims to help the company regain compliance with Nasdaq's minimum bid price requirement. Existing ADS holders will automatically see their ADSs exchanged, while fractional entitlements will be aggregated and sold for cash distribution. The company anticipates that this change will lead to a proportional increase in ADS trading price, though it cannot guarantee this outcome. The underlying ordinary shares will not be affected by the ratio change.
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