Cell and Gene Therapy
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Pharmaceutical
Shares in Swiss pharma giant Novartis shot up by 2.5% in Wednesday morning’s trading after the company presented its first quarter financial results. 24 April 2019
Biotechnology
Positive interim results from a Phase III trial evaluating the effect of Novartis’ Zolgensma (onasemnogene abeparvovec-xioi; AVXS-101) in Type 1 spinal muscular atrophy (SMA) patients is expected to make the case stronger for its approval and also its long-term use as a mainstream treatment option. 23 April 2019
Pharmaceutical
Looking back as news last week, Gilead Sciences’ collaboration with start-up insitro on non-alcoholic steatohepatitis (NASH) was noteworthy. On the M&A front there was the news that Catalent is acquiring Paragon Biosciences aimed at the gene therapy sector, and Kiadis Pharma buying CytoSen Therapeutics also to expand into gene therapy. On the pricing front, Amgen announced that its newly US approved osteoporosis drug Evenity would carry an annual price tag of $21,900. Featured clinical trial news included new data from Novartis subsidiary AveXis on its spinal muscular atrophy (SMA) drug Zolgensma. 21 April 2019
Biotechnology
Shares in GenSight Biologics were 7% higher with an hour’s trading left on Wednesday. 17 April 2019
Biotechnology
AveXis late Tuesday announced that interim data from its Phase III STR1VE trial of Zolgensma (onasemnogene abeparvovec-xioi; AVXS-101) in spinal muscular atrophy (SMA) Type 1 showed prolonged event-free survival, an early and rapid increase in CHOP-INTEND scores and significant milestone achievement compared to untreated natural history, consistent with data from the pivotal Phase 1 START trial. 17 April 2019
Biotechnology
Advanced delivery technologies firm Catalent has entered into a definitive agreement to acquire Paragon Bioservices, a leading viral vector development and manufacturing partner for gene therapies. 16 April 2019
Pharmaceutical
While there were no massive deals in March to rival those announced earlier in the year, there were still some notable deals announced. 4 April 2019
Biotechnology
Just a year after being acquired in an $8.7 billion deal by Swiss pharma giant Novartis, AveXis has signed an agreement to purchase an advanced biologics therapy manufacturing campus in Longmont, Colorado, USA. 2 April 2019
Pharmaceutical
The March 25-28 2019 meeting of the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) was held for the first time in its new post-Brexit offices in Amsterdam, Netherlands, but came back with only recommendations for one new initial marketing authorization and just three extensions of indication. 29 March 2019
Biotechnology
Thermo Fisher Scientific has reached a definitive agreement to acquire Brammer Bio, a leader in viral vector manufacturing for gene and cell therapies, with the news nudging its share 1.26% higher to $267.13 in early afternoon trading on Monday. 26 March 2019
Biotechnology
US biotech firm bluebird bio on Friday officially opened its first wholly-owned manufacturing facility in Durham, North Carolina, that will produce lentiviral vector for the company’s investigational gene and cell therapies. 25 March 2019
Pharmaceutical
The flurry of pharma/biotech M&A deals since the start of 2019 continues into March, with Biogen announcing its $877 million deal to acquire Nightstar Therapeutics. The decision last week of US Food and Drug Administration Commissioner Scott Gottlieb – seen as one of the most proactive heads of the FDA - to quit the post after just two years came as something of a surprise. Significant regulatory news last week included FDA approval for Johnson & Johnson subsidiary Janssen’s ketamine-based antidepressant Spravato. On the research front, Allergan’s hopes for its depression candidate rapastinel suffered a setback in the latest trial data revealed on Wednesday. 10 March 2019
Biotechnology
Following mixed results in a clinical study, Alnylam Pharmaceuticals lost ground in pre-market trading on Wednesday, ticking down nearly 3% in early morning trading. 6 March 2019
Biotechnology
French life sciences investment group Seventure Partners is already halfway to reaching its goal of raising 200 million euros ($227 million) for its microbiome-focused Health for Life Capital II fund. 6 March 2019
Biotechnology
The growing interest in gene therapies was further confirmed by US Biotech major Biogen, which has reached an agreement to acquire a UK-based clinical-stage gene therapy company developing treatments for rare inherited retinal diseases, at a price of $25.50 per share in cash. 4 March 2019
Pharmaceutical
“Everyone in the ecosystem recognizes that there's going to a lot of innovations coming forward in the next years and those need to get to patients.” 3 March 2019
Pharmaceutical
HIV/AIDS focused company ViiV Healthcare has entered a five-year collaboration with Radboud university medical center (Radboudumc) in Nijmegen, the Netherlands, providing dedicated funding to enhance the HIV-specific capability of the Human Functional Genomics Project (HFGP). 26 February 2019
Biotechnology
TC BioPharm, a Scottish biotech developing a platform of CAR-T immuno-oncology products which involve expansion of gamma-delta T cells, has announced a collaboration crossing the Irish sea. 25 February 2019
Biotechnology
Spark Therapeutics saw its shares rocket 120.62% to $113.75 in pre-market trading this morning, after it was revealed that it had received a takeover bid. 25 February 2019
Biotechnology
French gene therapy company GenSight Biologics was 32% down on its value at the end of last week by Wednesday lunchtime, the firm having reported results on its lead product. 6 February 2019
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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