Cell and Gene Therapy
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Biotechnology
Lumevoq (lenadogene nolparvovec) has been granted Promising Innovative Medicine (PIM) designation by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA). 7 September 2021
Biotechnology
Japanese pharma major Astellas Pharma has voluntarily paused screening and dosing of additional participants in its ASPIRO clinical trial evaluating AT132 in patients with X-linked myotubular myopathy (XLMTM). 1 September 2021
Biotechnology
These are billed as being exciting times for cell and gene therapies, but seasoned observers of the pharmaceutical industry would be forgiven for taking that billing with a pinch of salt. 31 August 2021
Biotechnology
Shares of Belgian cell therapy developer Bone Therapeutics tumbled 34.6% 1.73 euros by early afternoon trading, after the company announced that the Phase III knee osteoarthritis study with its enhanced viscosupplement JTA-004 did not meet the primary and consequently the key secondary endpoints. 30 August 2021
Biotechnology
Shares in Swiss pharma company Novartis were 1% lower by lunchtime on Tuesday following news of a setback for Kymriah (tisagenlecleucel), the first approved CAR-T cell therapy. 24 August 2021
Pharmaceutical
The UK government wants to expand its scientific partnership with Brazil’s research organization Fiocruz, especially in fields such as genomic sequencing, declared the minister counsellor of the British Embassy in Brazil, Melanie Hopkins, during a meeting with Fiocruz president Nísia Trindade Lima. 23 August 2021
Biotechnology
Adding to US regulatory clearance in March this year, US pharma major Bristol Myers Squibb has now won conditional approval for its a first-in-class B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy Abecma (idecabtagene vicleucel; ide-cel) from the European Commission. 19 August 2021
Biotechnology
Among last week’s notable news, US biotech firm bluebird bio revealed a double whammy on Wednesday, when it announced a US Food and Drug Administration clinical hold on its gene therapy Skysona (eli-cel) as well as its decision to pull out of Europe because of difficulties in gaining reimbursement for its products. AstraZeneca and partner FibroGen suffered a setback with the FDA issuing a complete response letter (CRL) on their anemia of chronic kidney disease candidate roxadustat. On the deal-making front, US biotech Seagen last week announced a licensing agreement with China’s RemeGen to develop disitamab vedotin as a treatment for various cancers, worth up to $2.2 billion. mRNA specialist Moderna released new data showing its COVID-19 vaccine Spikevax generated neutralizing antibodies against variants. 15 August 2021
Biotechnology
Mustang Bio, a cell & gene therapy specialist based in Massachusetts, USA, has executed an exclusive license agreement with the prestigious Mayo Clinic, related to its CAR-T cell technology. 13 August 2021
Biotechnology
The US Food and Drug Administration (FDA) has recently lifted a clinical hold on the spinal muscular atrophy (SMA) drug OAV-101, an intrathecal (IT) formulation of Zolgensma (onasemnogene abeparvovec). 11 August 2021
Biotechnology
Yet another public listing via special purpose acquisition company (SPAC) demonstrates the growing popularity of the approach, as an alternative to the traditional initial public offering. 11 August 2021
Biotechnology
The Parkinson’s disease (PD) market is expected to almost triple in sales in the seven major markets (7MM: The USA, France, Germany, Spain, Italy, the UK, and Japan) by 2029, rising from $4 billion in 2021 to $11.5 billion in 2029 at an impressive 12.6% compound annual growth rate (CAGR). 11 August 2021
Biotechnology
US biotech bluebird bio yesterday reported financial results and business highlights for the second quarter ended June 30, 2021, which failed to impress investors, so the stock tumbled 27.5% to $18.16 by close of trading. 10 August 2021
Biotechnology
US biotech BioMarin Pharmaceutical recently presented positive one-year results from its GENEr8-1 Phase III trial, in which adults with severe hemophilia A are treated with the gene therapy Roctavian (valoctocogene roxaparvovec). 9 August 2021
Biotechnology
The Kite unit of US biotech major Gilead Sciences has signed a collaboration and license agreement with Appia Bio, an early-stage biotech, to research and develop HSC-derived cell therapies directed toward hematological malignancies. 5 August 2021
Biotechnology
Swiss drugmaker Novartis has announced that the US Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) trials for spinal muscular atrophy (SMA) patients can go ahead. 3 August 2021
Biotechnology
USA-based cell and gene therapy specialist Mustang Biotech’s shares edged up 1.4% to $2.83 in after-hours trading, following the0 announcement that the European Medicines Agency has granted Priority Medicines (PRIME) designation to MB-107, its lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency (XSCID) in newly diagnosed infants, also known as bubble boy disease. 3 August 2021
Biotechnology
Minovia Therapeutics and Japan’s Astellas Pharma on Friday announced a worldwide strategic collaboration and license agreement for the research, development, and commercialization of novel cell therapy programs for diseases caused by mitochondrial dysfunction. 2 August 2021
Biotechnology
Last week Denali Therapeutics presented early-stage clinical data on its Hunter syndrome candidate DNL310 that did not go down too well with investors. On the deal-making front, US biotech bluebird bio reached agreement with National Resilience to develop next generation cell therapies that will earn it $100 million, while Japan’s PeptiDream struck another mega-bucks licensing accord last week, this time with US RNAi specialist Alnylam worth a potential $2.2 billion. Also, last Tuesday, biotech giant Amgen announced it is acquiring Teneobio in order to help strengthen its portfolio. 1 August 2021
Biotechnology
The UK genomics sector has released a report detailing the strength of research in this area, highlighting what it sees as a broad range of investment opportunities. 30 July 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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