RNA editing: emerging from CRISPR’s shadow
Bio Pharma Dive
OCTOBER 22, 2024
Early study data from Wave Life Sciences suggests how editing RNA may yield viable medicines. Large and small drugmakers say such results are just the start.
Startup AIRNA emerges from stealth to bring RNA editing to a rare disease
Bio Pharma Dive
SEPTEMBER 19, 2023
AIRNA is one of several biotechs to debut plans for target alpha-1 antitrypsin deficiency in recent years, with competitors such as AlveoGene, Wave Life Sciences and Arrowhead Pharmaceuticals.
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GSK and Wave Life Sciences partner to develop oligonucleotide therapeutics
Pharmaceutical Technology
DECEMBER 14, 2022
GSK has entered a strategic partnership with Wave Life Sciences to progress the discovery and development of oligonucleotide therapies for new genetic targets. An RNA editing therapy, WVE-006, could act on AATD’s liver and lung manifestations. According to the deal, GSK will make an upfront payment of $170m to Wave.
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Pharmas back Accent’s plan to make RNA-targeting cancer drugs
Bio Pharma Dive
JANUARY 23, 2024
J&J and Bristol Myers joined the latest funding round for the startup, which was led by the newly launched Mirae Asset Capital Life Science.
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Key Trends in the Life Sciences to Look Forward to in 2022
XTalks
DECEMBER 22, 2021
If the past year is anything to go by, then 2022 will also be a year marked by continued innovations in the life sciences. Here is a look at some life science trends to expect in the coming year, including key themes and topics in the pharmaceutical, biotechnology and medical device industries to keep an eye out for in 2022.
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Looking at Women’s Leadership in Life Science Industries on International Women’s Day
XTalks
MARCH 8, 2022
On International Women’s Day, Xtalks is celebrating women’s leadership in the life sciences by highlighting some of the female leaders at the forefront of scientific discovery, as well as the continuing challenges of attaining more equitable representation. Challenges in Women’s Leadership.
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Eli Lilly Pursues RNA Editing in New Partnership with ProQR
XTalks
SEPTEMBER 13, 2021
With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare genetic diseases with a focus on blinding disorders of the retina.
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Wave surges on first RNA editing data in humans
pharmaphorum
OCTOBER 16, 2024
Shares in Wave Life Sciences rose by 74% after it reported clinical data with a pioneering RNA-editing therapy for alpha-1 antitrypsin deficiency (AATD)
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Alnylam Files Lawsuits Against Pfizer and Moderna Over Patent Infringement of RNA Delivery Technology
XTalks
MARCH 21, 2022
Credited as a pioneering RNA interference (RNAi) therapeutics company, Alnylam Pharmaceuticals is taking Pfizer and Moderna to court, claiming that the companies’ use of the lipid nanoparticle (LNP)-based RNA delivery technology in their mRNA COVID-19 vaccines infringes on a patented technology.
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BioTuring enters collaboration to enable detailed single-cell RNA research
pharmaphorum
JANUARY 19, 2022
The collaboration aims to “close the gap between single-cell RNA sequencing wet-lab services and single-cell data analysis solutions,” which will allow scientists to study the finest details that can be hard to access. Research Instruments is the main distributor for genomic and life science research products in the region.
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FDA and CDC Update Guidelines Amid Omicron + Life Science Trends for 2022 – Xtalks Life Science Podcast Ep. 42
XTalks
JANUARY 5, 2022
Ayesha also discussed some of the predicted trends in the life sciences for 2022. These include the continued promise of RNA-based vaccines as well as therapeutics based on RNA editing approaches for the treatment of various conditions including rare genetic diseases.
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mRNA Disruptor Goes Public in $1.5 Billion SPAC Deal
BioSpace
AUGUST 9, 2021
Messenger RNA has become the life sciences hero of the decade. The lynchpin in the fight against COVID-19, companies are taking mRNA technology to new heights.
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Wave claims first clinical RNA editing title in rare genetic disease
BioPharma Reporter
OCTOBER 17, 2024
Wave Life Sciences reports that it has successfully carried out RNA editing in two patients with alpha-1 antitrypsin deficiency, the first time this kind of clinical RNA editing has been completed in humans according to the company.
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Listen: How blockbusters get made, new vaccines for RSV, and mRNA’s Q score
STAT News
JANUARY 19, 2023
Does science need billionaires? And do normal people care about messenger RNA? Journalist Nathan Vardi joins us to talk about his new book delving into the race to develop the lifesaving cancer drug now called Imbruvica, involving a Scientologist CEO and secretive investor seeking redemption after the worst trade of his life.
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Sartorius upscales gene therapy prowess with $2.6bn Polyplus acquisition
Pharmaceutical Technology
APRIL 3, 2023
The deal will see Polyplus join the German life science group’s portfolio allowing the latter to leverage expertise in transfection reagents and plasmid DNA for gene therapy. Sartorius , through its French listed sub-group Sartorius Stedim Biotech , has signed an agreement to acquire Polyplus for €2.4bn ($2.6bn).
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Global Roundup: ProQR Licenses Axiomer RNA Editing Platform to Eli Lilly
BioSpace
SEPTEMBER 8, 2021
Biopharma and life sciences companies from across the globe provide updates on their pipelines and businesses.
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Jupiter Neurosciences Makes Way in CNS Treatments with $11M IPO
XTalks
DECEMBER 4, 2024
CAMP4’s RNA-based therapies focus on genetic diseases like urea cycle disorders, while Rapport’s small molecules aim to address epilepsy, pain and bipolar disorder. Jupiter joins a wave of biotech IPOs this year, stepping into a competitive space while broadening treatment options for CNS patients.
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Donidalorsen Shows Sustained HAE Attack Reduction According to New Trial Data
XTalks
FEBRUARY 24, 2025
Carlsbad, California-based Ionis Pharmaceuticals announced it will be sharing promising new trial data for its RNA-targeted prophylactic treatment donidalorsen for hereditary angioedema (HAE) at the 2025 American Academy of Allergy, Asthma & Immunology (AAAAI)/World Allergy Organization (WAO) Joint Congress in San Diego, California.
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Eli Lilly’s New Partnership to Develop RNA Editing Therapies + Study Shows How Many Steps a Day Offers Benefit – Xtalks Life Science Podcast Ep. 28
XTalks
SEPTEMBER 15, 2021
With RNA being the popular new “it” molecule in genetic research, Ayesha discusses Eli Lilly’s new partnership with biotech company ProQR to develop RNA-based therapeutics in this episode. billion deal will see Lilly leverage ProQR’s proprietary RNA editing platform to develop targets of interest.
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Pfizer’s New RSV Vaccine TV Commercial Calls for Protecting Moments
XTalks
NOVEMBER 15, 2024
XTALKS WEBINAR: Solutions for Vaccine Innovation and Gene Therapy: Unlocking the Power of RNA Live and On-Demand: Tuesday, December 17, 2024 , at 9am EST (3pm CET / EU-Central) Register for this free webinar to explore how next-generation RNA technologies can provide effective solutions for vaccine innovation and gene therapy.
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Exact Sciences and Katie Couric’s Colon Cancer Awareness TV Ad + Alnylam Sues Pfizer and Moderna Over Patent Infringements – Xtalks Life Science Podcast Ep. 53
XTalks
MARCH 23, 2022
Moderna hit back at Alnylam, accusing it of “blatant opportunism” and saying that it has worked for years on a different version of the RNA delivery technology. Read the full articles here: Alnylam Files Lawsuits Against Pfizer and Moderna Over Patent Infringement of RNA Delivery Technology.
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Wave Life Sciences Announces Proof-of-Concept Preclinical Data for ADAR Editing Program in Alpha-1 Antitrypsin Deficiency
BioTech 365
JUNE 2, 2021
Wave Life Sciences Announces Proof-of-Concept Preclinical Data for ADAR Editing Program in Alpha-1 Antitrypsin Deficiency Wave Life Sciences Announces Proof-of-Concept Preclinical Data for ADAR Editing Program in Alpha-1 Antitrypsin Deficiency First proof-of-concept in vivo data for RNA editing using endogenous … Continue reading →
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Solid Biosciences partners with Phlox for cardiac disease therapies
Pharmaceutical Technology
JANUARY 12, 2023
The collaboration will use the vector biology, manufacturing capabilities and the drug development experience of Solid Biosciences and Phlox Therapeutics’ knowledge in genetic cardiomyopathies and RNA therapies. Solid Biosciences stated that the partnership boosts its scientific capabilities along with commercial potential in cardiac therapy.
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LabCorp’s Innovative High-Speed COVID-19 Test Gets Emergency Use Authorization from the FDA
XTalks
OCTOBER 7, 2020
LabCorp – a leading global life sciences company based in Burlington, North Carolina that focuses on advancing health and guiding patient care decisions – announced that it is the first commercial laboratory to receive an EUA from the FDA for the new, innovative, high-speed test.
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Complex Medicines: A multi-billion-pound addition to the UK life sciences industry
pharmaphorum
JULY 19, 2021
Drugs harnessing the potential of modified RNA, antisense and siRNA oligonucleotides, antibody–drug conjugates and nanomedicines will become as important as today’s ‘small molecule’ drugs, reaching potential market values into the hundreds of billions – if the potential can be realised through innovation support and industry collaboration.
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Lab to jab in 100 days: manufacturing flexibility for future rapid responses
Pharmaceutical Technology
NOVEMBER 22, 2022
The mission was first put forward during the UK’s G7 Presidency of June 2021, with support from representatives of the life sciences industry. Messenger RNA vaccines contain nucleic acids that code for a specific protein, or target antigen, related to a virus or disease. The UK Government is behind this effort.
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Pioneering mRNA Therapies for Lung Diseases
XTalks
SEPTEMBER 25, 2024
Rudolph and Professor Christian Plank with the goal of establishing messenger RNA (mRNA) as a novel drug modality for lung diseases such as asthma and rare pulmonary conditions like primary ciliary dyskinesia (PCD) and pulmonary alveolar proteinosis. Subscribe to the Xtalks Life Science Podcast to never miss a new episode.
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World’s First RSV Vaccine + First Novel Treatment for ALS – Xtalks Life Science Podcast Ep. 109
XTalks
MAY 10, 2023
Subscribe to the Xtalks Life Science Podcast to never miss a new episode. Read the full articles here: Arexvy Becomes World’s First RSV Vaccine Qalsody (Tofersen) Becomes First-of-Its-Kind Treatment for ALS The weekly podcast is available for streaming every Wednesday on Spotify , Apple Music and wherever you stream your podcasts.
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UK prepares for decision on AZ/Oxford vaccine as COVID cases soar
pharmaphorum
DECEMBER 24, 2020
AZ’s vaccine, dubbed AZD1222, is stored at normal refrigerator temperatures while the Pfizer/BioNTech and Moderna shots need to be kept at around -70C and -20C respectively to maintain the integrity of their RNA structure. I would expect some news pretty shortly, I doubt we will make Christmas now but just after Christmas I would expect.
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The Silent Mutations That Gave Coronavirus a Selective Advantage
XTalks
OCTOBER 21, 2020
In addition, Berrio and colleagues discovered other novel targets of positive selection, namely within genes encoding Nsp4 and Nsp16, which are viral enterotoxins important for the stability of the RNA structure of the virus. Moreover, Berrio says the changes likely affected the folding and function of viral RNA.
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Combining the Powers of Single Cell Sequencing and AI in Understanding Disease Biology and Drug Development
XTalks
AUGUST 5, 2020
In recent webinars by Genuity Science, formerly known as WuXi NextCODE, experts from the biotech and pharma industries spoke about leveraging the power of single cell RNA sequencing platforms and solutions in conjunction with machine learning technologies such as AI in cell biology and disease research.
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The siRNA STP705 by Sirnaomics Has Promise for Treating in situ Squamous Cell Carcinoma (isSCC)
XTalks
JUNE 20, 2022
Small interfering RNAs (siRNAs) are a promising class of therapeutic modalities that involve a double-stranded RNA molecule (20 to 25 bp long) which can target and cease the translation of a disease-related mRNA. 1 (transforming growth factor beta1) and the enzyme COX-2 (cyclooxygenase-2) is strongly linked with SCC development.
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Amvuttra RNAi Therapeutic Wins FDA Approval for Rare Genetic Protein Disorder
XTalks
JUNE 20, 2022
Alnylam Pharmaceuticals, a leading RNA interference (RNAi) therapeutics biopharmaceutical company, announced it received approval from the US Food and Drug Administration (FDA) for its RNAi therapeutic Amvuttra (vutrisiran) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (ATTR) amyloidosis in adults.
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Why Proviral DNA Drug Resistance Testing is Useful in Guiding Treatment Decisions for HIV-1 Patients
XTalks
APRIL 29, 2021
Compared to standard plasma HIV-1 RNA drug resistance profiling, which requires a viral load of at least 500 copies per mL, proviral HIV-1 testing can be performed on samples with much lower virion counts of <50 copies per mL (below the detectable limit in RNA testing). Proviral DNA Genotyping.
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LIB Therapeutics’ Cholesterol Lowering Med Lerodalcibep Receives FDA BLA Acceptance
XTalks
FEBRUARY 12, 2025
Current PCSK9 inhibitors on the market include Regenerons monoclonal antibody Praluent (alirocumab), Amgens Repatha (evolocumab) and Novartiss Leqvio (inclisiran), which is a small RNA interference (siRNA) therapy designed to lower LDL-C.
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Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data
XTalks
OCTOBER 26, 2021
Givlaari reduces ALAS1 levels through RNA interference-mediated gene silencing, which targets ALAS1 mRNA. Experts say gene silencing through RNA interference holds great promise in the targeting of diseases ranging from cancer to Alzheimer’s and cardiovascular disease. This leads to lowering of ALA and PBG levels.
New HCV Test Enables Diagnosis and Treatment in a Single Visit
XTalks
JULY 10, 2024
Unlike traditional testing methods that require samples to be sent to central laboratories, this test utilizes a blood sample from the fingertip to detect HCV RNA, delivering results in approximately one hour. “This is the first HCV RNA detection technology sensitive enough for active case finding at the point of care.
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Vutrisiran Reduces Heart Risks for ATTR-CM Patients in Alnylam’s HELIOS-B Trial
XTalks
SEPTEMBER 4, 2024
Alnylam Pharmaceuticals announced promising results from its HELIOS-B Phase III clinical trial evaluating vutrisiran, an investigational RNA interference (RNAi) therapeutic for treating transthyretin amyloidosis with cardiomyopathy (ATTR-CM). As of March 2024, the FDA has approved six small interfering RNA (siRNA) therapies.
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Winship Cancer Institute of Emory University Joins Caris’ Precision Oncology Alliance
The Pharma Data
OCTOBER 19, 2020
20, 2020 /PRNewswire/ — Caris Life Sciences® , a leading innovator in molecular science focused on fulfilling the promise of precision medicine, announced today that Winship Cancer Institute of Emory University (Winship) has joined Caris’ Precision Oncology Alliance (The Alliance/POA). .
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Eli Lilly Announces Nearly $1.5 Billion Investment into New Manufacturing Facilities in North Carolina and Ireland
XTalks
FEBRUARY 3, 2022
Related: Eli Lilly Pursues RNA Editing in New Partnership with ProQR. In the news release from IDA outlining Lilly’s new Irish manufacturing plant, the agency said Ireland has made a name for itself as a life sciences and biopharma manufacturing hub. This includes the company’s foray into RNA technologies such as RNA editing.
Discovery of MicroRNA Genetic Regulation Leads American Scientists to Win 2024 Nobel Prize
XTalks
OCTOBER 8, 2024
In a statement , the Nobel Assembly said the laureates discovered a new class of non-coding RNA molecules that play a crucial role in gene regulation. To activate these instructions, cells create a copy called messenger RNA (mRNA), which exits the nucleus and directs the cell’s machinery to produce a specific protein.
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Wainua (Eplontersen) Now FDA-Approved for Rare Disease ATTRv-PN
XTalks
JANUARY 3, 2024
Amvuttra: Alnylam’s Similar Treatment on the Market In 2022, Alnylam, a US-based biopharma company with expertise in RNA interference therapeutics for genetically defined diseases, received FDA approval for its drug Amvuttra (vutrisiran) for the treatment of hATTR.
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Medicines Discovery Catapult awarded funding to establish Cheshire & Warrington medicines powerhouse
pharmaphorum
AUGUST 11, 2020
MDC already has established working relationships with many life science organisations across the region and provides input into the LEP’s regional life sciences strategy. The funding will be used to create a regional consortium and develop the full stage bid.
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Amgen acquires Teneobio for $2.5B; Signifier scores $35M; Biogen-Ionis’ drug meets a goal; AbbVie hits go on $1B re-upped Calico deal
Delveinsight
JULY 29, 2021
An antisense treatment, BIIB080 potentially aims at microtubule-linked protein tau (MAPT) messenger RNA (mRNA) and impedes tau protein production. AbbVie hits go on USD 1 Billion re-upped Calico deal as Google’s life science spin-out continues I-O, Neuropush.
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