Cell and Gene Therapy
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Biotechnology
US biotech major Gilead Sciences yesterday posting financials for second-quarter 2021, showing that revenues of $6.2 billion increased 21% compared to the same period in 2020. 30 July 2021
Biotechnology
US biotech bluebird bio edged up 2% in early trading yesterday, after it announced an agreement with National Resilience to accelerate the early research, development, and delivery of next-generation cell therapies. 29 July 2021
Biotechnology
Last week saw the announcement of two large licensing agreements. First, Gilead Sciences’ subsidiary Kite entered into a deal with BioNTech, whereby the German biotech, mostly in the news due to its COVID-19 vaccine developed with Pfizer, has acquired an R&D platform and clinical manufacturing facility in Maryland. The second deal was US pharma giant Pfizer signing an agreement with Arvinas for rights to the latter’s breast cancer candidate, ARV-471, that could cost it more than $2 billion. On the regulatory front, US biotech bluebird bio gained regulatory approval from the European Commission for its gene therapy Skysona. Also, Albireo received approval from both the US Food and Drug Administration and the European Medicines for its rare liver disease drug Bylvay. 25 July 2021
Biotechnology
The European Medicines Agency’s ’s human medicines committee, CHMP, has endorsed findings of a review which concluded that there is no evidence Zynteglo causes a blood cancer known as acute myeloid leukemia (AML). 23 July 2021
Biotechnology
Cancer-focused cell therapy company ImmPACT Bio has announced its merger with Californian biotech Kalthera. 22 July 2021
Biotechnology
Following a positive recommendation from the European Medicines Agency’s human medicines committee (CHMP) in May, the European Commission (EC) has granted marketing authorization of Skysona (elivaldogene autotemcel, Lenti-D), developed by US biotech bluebird bio. 21 July 2021
Biotechnology
Two doses of Ryoncil (remestemcel-L) at days 3-5 conferred durable survival benefit through at least 90 days in a pre-specified subgroup of patients under age 65, according to Australian biotech Mesoblast. 19 July 2021
Biotechnology
German biotech BioNTech has entered into a purchase agreement to acquire Gilead Sciences’ subsidiary Kite’s solid tumor neoantigen T cell receptor (TCR) R&D platform and clinical manufacturing facility in Gaithersburg, Maryland. 19 July 2021
Biotechnology
England’s health technology assessor, the National Institute for Health and Care Excellence (NICE), has dealt a blow to Orchard Therapeutics’ reimbursement goals for atidarsagene autotemcel. 12 July 2021
Biotechnology
Belgian cell therapy company Bone Therapeutics, which is addressing unmet medical needs in orthopedics and other diseases, has appointed Anne Leselbaum as chief medical officer (CMO), effective August 23, 2021. 12 July 2021
Biotechnology
The European Medicines Agency’s Pharmacovigilance Risk Assessment Committee (PRAC) held its July meeting this past week, and today reported on safety issues with a leukemia drug and four COVID-19 vaccines. 9 July 2021
Biotechnology
The UK’s reimbursement agency has recommended the use of Novartis’ Zolgensma (onasemnogene abeparvovec) for spinal muscular atrophy (SMA) in babies. 8 July 2021
Biotechnology
When the US regulator said in December it could not complete its review of a submission for heart med inclisiran, a potential blockbuster, it came as something of a shock. 7 July 2021
Biotechnology
Australian off-the-shelf cell therapy specialist Mesoblast saw its shares lifted a few percentage points on Thursday, following encouraging news on the regulatory front. 2 July 2021
Biotechnology
Parisian biotech GenSight Biologics saw its stock drop 10% in value on Wednesday, after the gene therapist announced more confusing results from its Phase III program. 1 July 2021
Biotechnology
UK and USA-based clinical-stage gene therapy specialist Orchard Therapeutics as entered into a strategic collaboration with Dutch firm Pharming Group to research, develop, manufacture and commercialize OTL-105. 1 July 2021
Biotechnology
Japan’s FUJIFILM Corporation today announced a 90 billion yen ($850 million) investment package to accelerate the growth of its core Bio Contract Development and Manufacturing Organization (CDMO) subsidiary, Fujifilm Diosynth Biotechnologies. 30 June 2021
Biotechnology
UK pharma major GlaxoSmithKline last Wednesday hosted a much-anticipated investor update, where it showcased its sales aims and R&D pipeline, ahead of splitting out its consumer health care business. Dutch gene therapy company uniQure released positive Phase IV data on its hemophilia B candidate etranacogene dezaparvovec. The US Food and Drug Administration issued Breakthrough Therapy designations for two Alzheimer’s disease candidates, first for Biogen and Eisai’s lecanemab and, a day later, Eli Lilly’s donanemab. Meanwhile, US biotech Incyte suffered a setback on Thursday, as an FDA advisory committee voted that further data was required prior to an approval decision on its immuno-oncology drug retifanlimab. 27 June 2021
Biotechnology
At its June meeting, the European Medicines Agency’s human medicines committee (CHMP) recommended six novel medicines for approval, and issued a negative opinion on one. 26 June 2021
Biotechnology
The Fabry disease (FD) market in the USA and Japan will see significant expansion between 2020 and 2030, as total sales in the two markets are set to rise from $820.15 million in 2020 to $985.81 million in 2030, at a compound annual growth rate (CAGR) of 1.9%. 25 June 2021
Biotechnology
US clinical-stage biotech Vyriad has entered into a strategic collaboration with Swiss pharma giant Novartis to discover and develop in vivo chimeric antigen receptor (CAR) T-cell therapies. 21 November 2024
Biotechnology
Australia’s CSL Limited plans to shut down its Californian R&D facility focused on cell and gene therapies by January 2025, signaling a shift away from ex vivo lentiviral-based technology. 19 November 2024
Biotechnology
Privately-held UK firm Quell Therapeutics, which positions itself as a pioneer in developing engineered T-regulatory (Treg) cell therapies for serious medical conditions driven by the immune system, has announced that AstraZeneca has selected a candidate to progress in the type 1 diabetes (T1D) Treg cell therapy program. 18 November 2024
Biotechnology
A Feature analyzing M&A, licensing and partnerships in gene editing as CRISPR therapeutics enter the market and present new opportunities for pharma growth. 15 November 2024
Biotechnology
The US Food and Drug Administration (FDA) has granted accelerated approval of PTC Therapeutics’ gene therapy for the treatment of AADC (aromatic L-amino acid decarboxylase) deficiency, making it the first-ever gene therapy approved in the USA that is directly administered to the brain. 14 November 2024
Biotechnology
Neurogene has announced positive interim clinical data in the first four participants in the low-dose cohort of its ongoing Phase I/II open-label trial designed to evaluate NGN-401 gene therapy for the treatment of female pediatric patients with Rett syndrome. 13 November 2024
Biotechnology
Sino-American oncology company BeiGene has announced third-quarter 2024 financials, showing the total revenues was $1,002 million, up 28% compared to $781 million in the same period of 2023. 13 November 2024
Biotechnology
Sarepta Therapeutics has announced it will cease development of SRP-5051, an experimental drug for Duchenne muscular dystrophy, citing safety issues, feedback from the Food and Drug Administration, and the shifting Duchenne treatment landscape. 11 November 2024
Biotechnology
Asklepios BioPharmaceutical (AskBio) today announced that AB-1003 (also known as LION-101) has received rare pediatric disease designation and orphan-drug designation from the US Food and Drug Administration (FDA) for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). 7 November 2024
Biotechnology
The global market for Duchenne muscular dystrophy (DMD) therapeutics is projected to grow by $4.4 billion between 2024 and 2028, fueled by artificial intelligence and strong demand for new treatments. 7 November 2024
Biotechnology
Ring Therapeutics, a biotech based in Massachusetts, USA, has announced strategic partnerships with Singapore’s Agency for Science, Technology, and Research (A*STAR) and the Singapore Eye Research Institute (SERI). 5 November 2024
Biotechnology
The global DNA synthesis market will grow by $6.5 billion over the next five years, according to a new report from Technavio. 29 October 2024
Biotechnology
Last week Vertex Pharmaceuticals released positive Phase III results for its suzetrigine for moderate-to-severed pain. Belgium’s UCB revealed that Swiss pharma giant Roche was terminating its collaboration on the Alzheimer’s candidate bepranemab. The UK’s health technology assessor the Institute for Health and Clinical Excellence (NICE) declined to recommend a second Alzheimer’s treatment, this time Eli Lilly’s Kisunla. Also of note, Lyell Immunopharma announced its planned acquisition of ImmPACT Bio and reprioritization of its own clinical pipeline. 27 October 2024
Biotechnology
California, USA-based Lyell Immunopharma (Nasdaq: LYEL) has entered into a definitive agreement to acquire privately-held US CAR-T company ImmPACT Bio. 26 October 2024
Biotechnology
Intellia Therapeutics has announced Phase II results for its CRISPR-based therapy, NTLA-2002, aimed at providing a one-time treatment for hereditary angioedema (HAE). 25 October 2024
Biotechnology
US artificial intelligence (AI) to gene therapy focussed biotech Dyno Therapeutics today announced its second research collaboration with Roche to develop next-generation adeno-associated virus (AAV) vectors for gene therapies targeting neurological diseases. 24 October 2024
Biotechnology
Ocuphire Pharma has announced its acquisition of Opus Genetics, a clinical-stage gene therapy company focused on treating inherited retinal diseases. 24 October 2024
Biotechnology
Editas Medicine has announced plans to seek a partner or buyer for its lead CRISPR program, reni-cel, as the company continues to shift its strategy. 23 October 2024
Biotechnology
Shares in Sangamo Therapeutics surged around 40% on Tuesday, following the announcement that the US Food and Drug Administration has granted the company a faster path to approval for its gene therapy ST-920 (isaralgagene civaparvovec) for Fabry disease. 23 October 2024
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