LNP

CRISPR Therapeutics Provides Business Update and Reports Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Wednesday, February 21, 2024

R&D Expenses: R&D expenses were $95.1 million for the fourth quarter of 2023, compared to $103.6 million for the fourth quarter of 2022.

Key Points: 
  • R&D Expenses: R&D expenses were $95.1 million for the fourth quarter of 2023, compared to $103.6 million for the fourth quarter of 2022.
  • G&A Expenses: General and administrative expenses were $16.5 million for the fourth quarter of 2023, compared to $21.2 million for the fourth quarter of 2022.
  • Collaboration Expense: Collaboration expense, net, was $20.0 million for the fourth quarter of 2023, compared to $6.8 million for the fourth quarter of 2022.
  • Net Income (Loss): Net income was $89.3 million for the fourth quarter of 2023, compared to a net loss of $110.6 million for the fourth quarter of 2022.

ReCode Therapeutics Announces First Participants Dosed in a Phase 1 Healthy Volunteer Clinical Study of Inhaled mRNA-Based Genetic Medicine, RCT2100, for the Treatment of Cystic Fibrosis

Retrieved on: 
Wednesday, February 21, 2024

“While progress has been made for many people with CF, those who do not benefit from existing modulators report feeling left behind and continue to face profound physical and emotional hardship from the disease.

Key Points: 
  • “While progress has been made for many people with CF, those who do not benefit from existing modulators report feeling left behind and continue to face profound physical and emotional hardship from the disease.
  • The Phase 1 double-blind, placebo-controlled, first-in-human study is designed to assess the safety and tolerability of a single ascending dose of inhaled RCT2100 administered via nebulizer.
  • The study is being conducted in New Zealand and ReCode anticipates enrolling approximately 32 healthy adults who will receive a single dose of either placebo or RCT2100.
  • ReCode plans to submit an Investigational New Drug application (IND) to the U.S. Food and Drug Administration for a Phase 1 trial of RCT2100 in CF patients in the first half of 2024.

Almirall continues its progress to leadership in Medical Dermatology driven by strong growth in the European Dermatology business

Retrieved on: 
Monday, February 19, 2024

Net Sales of €895 MM represented year-on-year growth of 4%, driven by strong performance of the European Dermatology business.

Key Points: 
  • Net Sales of €895 MM represented year-on-year growth of 4%, driven by strong performance of the European Dermatology business.
  • Powered by our dedicated investments in launches and innovation, we achieved a 4% year-on-year growth in Net Sales, accounting for €895 MM, which was mainly driven by our European dermatology business.
  • Almirall has complemented its exciting pipeline and discovery efforts through strategic partnerships, adding long-term value to its medical dermatology portfolio.
  • The strategic focus on medical dermatology has positioned the company well for sustained growth and leadership in the medium term and beyond.

Intellia Therapeutics and ReCode Therapeutics Announce Strategic Collaboration to Develop Novel Gene Editing Therapies for Cystic Fibrosis

Retrieved on: 
Thursday, February 15, 2024

and MENLO PARK, Calif., Feb. 15, 2024 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, and ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, today announced a strategic collaboration to develop novel genomic medicines for the treatment of cystic fibrosis (CF).

Key Points: 
  • and MENLO PARK, Calif., Feb. 15, 2024 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, and ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, today announced a strategic collaboration to develop novel genomic medicines for the treatment of cystic fibrosis (CF).
  • Intellia will be responsible for the design of the editing strategy and research-grade components for the investigational therapies.
  • “This collaboration provides further validation of ReCode's SORT LNP platform to deliver diverse gene editing modalities to specific cells and tissues.
  • By combining our highly synergistic technologies and capabilities, we are excited about the potential to enable a faster path for next-generation gene editing therapeutics to CF patients.”

Intellia Therapeutics and ReCode Therapeutics Announce Strategic Collaboration to Develop Novel Gene Editing Therapies for Cystic Fibrosis

Retrieved on: 
Thursday, February 15, 2024

Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, and ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, today announced a strategic collaboration to develop novel genomic medicines for the treatment of cystic fibrosis (CF).

Key Points: 
  • Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, and ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, today announced a strategic collaboration to develop novel genomic medicines for the treatment of cystic fibrosis (CF).
  • Intellia will be responsible for the design of the editing strategy and research-grade components for the investigational therapies.
  • “This collaboration provides further validation of ReCode's SORT LNP platform to deliver diverse gene editing modalities to specific cells and tissues.
  • By combining our highly synergistic technologies and capabilities, we are excited about the potential to enable a faster path for next-generation gene editing therapeutics to CF patients.”

Sail Biomedicines Appoints Kerry Benenato, Ph.D., as Chief Platform Officer

Retrieved on: 
Wednesday, February 7, 2024

Sail Biomedicines, Inc. (“Sail”), a Flagship Pioneering company and leader in RNA-based programmable medicines, today announced that Kerry Benenato, Ph.D., has been appointed to the position of the role of Chief Platform Officer, replacing Stuart Milstein, Ph.D., who is leaving the company to pursue other opportunities.

Key Points: 
  • Sail Biomedicines, Inc. (“Sail”), a Flagship Pioneering company and leader in RNA-based programmable medicines, today announced that Kerry Benenato, Ph.D., has been appointed to the position of the role of Chief Platform Officer, replacing Stuart Milstein, Ph.D., who is leaving the company to pursue other opportunities.
  • Dr. Benenato brings to Sail a storied career in the development of mRNA products and product candidates.
  • “We are delighted to welcome Kerry to Sail and its Executive Leadership Team.
  • “The potential of Sail’s platform – combining industry-leading Endless RNA™ (eRNA™) technology with uniquely programmable deployment nanoparticles, accelerated by AI – is unparalleled within the biopharma industry.

Labor’s Newspoll lead unchanged since December as 62% support stage three changes

Retrieved on: 
Monday, February 5, 2024

A national Newspoll, conducted January 31 to February 3 from a sample of 1,245, gave Labor a 52–48 lead, unchanged since the previous Newspoll in mid-December.

Key Points: 
  • A national Newspoll, conducted January 31 to February 3 from a sample of 1,245, gave Labor a 52–48 lead, unchanged since the previous Newspoll in mid-December.
  • Primary votes were 36% Coalition (steady), 34% Labor (up one), 12% Greens (down one), 7% One Nation (steady) and 11% for all Others (steady).
  • Anthony Albanese’s net approval dropped one point to -9, while Peter Dutton’s net approval was down four points to -13.
  • His net approval is still well below zero, and hasn’t recovered to its level before the Voice referendum defeat.

Essential poll: 48–46 to Labor

  • In last week’s federal Essential poll, conducted January 24–28 from a sample of 1,201, Labor led by 48–46 including undecided (49–46 in December).
  • Labor has led by one-to-three points in all Essential polls since late October.
  • Primary votes were 34% Coalition (steady), 32% Labor (up one), 13% Greens (steady), 7% One Nation (up one), 2% UAP (steady), 7% for all Others (down two) and 5% undecided (steady).
  • Analyst Kevin Bonham said Labor would have about a 53–47 lead in this poll by 2022 election preference flows.

Morgan poll and a second Queensland byelection

  • In last week’s federal Morgan poll, conducted January 22–28 from a sample of 1,688, Labor led by 50.5–49.5, a two-point gain for the Coalition since the previous week.
  • Primary votes were 37.5% Coalition (up 1.5), 31% Labor (down 1.5), 13% Greens (up 0.5), 5.5% One Nation (up 0.5) and 13% for all Others (down one).
  • I covered the March 16 Queensland state byelection in Inala last fortnight.
  • A second Queensland byelection will also occur on March 16 after Ipswich West’s Labor member Jim Madden resigned to contest the Ipswich local government elections on March 16.

Biden wins 96% in South Carolina Democratic primary

  • At Saturday’s United States Democratic presidential primary in South Carolina, Joe Biden won 96.2% of the vote, Marianne Williamson 2.1% and Dean Phillips 1.7%.
  • This result makes it all but certain that Biden will be the Democratic presidential nominee.
  • In the Republican presidential contest, Donald Trump leads Nikki Haley nationally by 73.6–17.2 in the FiveThirtyEight aggregate.
  • The next important contest is the February 24 Republican primary in South Carolina, Haley’s home state.


Adrian Beaumont does not work for, consult, own shares in or receive funding from any company or organisation that would benefit from this article, and has disclosed no relevant affiliations beyond their academic appointment.

Thermo Fisher Scientific Receives GMP approval from the Italian Medicines Agency (AIFA) for its Manufacturing facility of RNA-Based Products in Monza, Italy

Retrieved on: 
Friday, January 19, 2024

Thermo Fisher Scientific, the world leader in serving science, has received Good Manufacturing Practice (GMP) approval from the Italian Medicines Agency (AIFA) allowing the company to manufacture RNA-based products at its Monza, Italy site.

Key Points: 
  • Thermo Fisher Scientific, the world leader in serving science, has received Good Manufacturing Practice (GMP) approval from the Italian Medicines Agency (AIFA) allowing the company to manufacture RNA-based products at its Monza, Italy site.
  • The approval and associated certification support increased accessibility to novel therapies for patients with difficult to treat conditions, marking a significant achievement within the Thermo Fisher Scientific network and for Italy as a whole.
  • “RNA technology is being explored for a variety of modalities, including rare diseases and cancer which have limited treatment options,” said Dan Herring, General Manager, Advanced Therapies, Pharma Services at Thermo Fisher Scientific.
  • With more than 30 years of experience manufacturing sterile injectables, biologics, and advanced therapies, Thermo Fisher has the capabilities and expertise required to help customers accelerate development and increase access to therapies for patients globally.

WestGene Spearheads Oncology Breakthroughs at the 3rd mRNA-Based Therapeutics Summit

Retrieved on: 
Wednesday, January 24, 2024

CHENGDU, China, Jan. 24, 2024 /PRNewswire/ -- Kicking off the New Year with remarkable achievements, Chengdu-based WestGene made a significant impact at the 3rd mRNA-Based Therapeutics Summit in Berlin, Germany.

Key Points: 
  • CHENGDU, China, Jan. 24, 2024 /PRNewswire/ -- Kicking off the New Year with remarkable achievements, Chengdu-based WestGene made a significant impact at the 3rd mRNA-Based Therapeutics Summit in Berlin, Germany.
  • The Summit, held January 23-25, is a key event in the field of mRNA drug development, attracting over 200 specialists from various parts of the world.
  • Founded by two visionary scientists, Dr. Wei Yuquan and Dr. Xiangrong Song, WestGene Biotech is dedicated to pioneering the field of mRNA therapeutics.
  • This project, along with its extensive pipeline, positions WestGene at the forefront of biopharmaceutical innovation and research.

WestGene Spearheads Oncology Breakthroughs at the 3rd mRNA-Based Therapeutics Summit

Retrieved on: 
Wednesday, January 24, 2024

CHENGDU, China, Jan. 24, 2024 /PRNewswire/ -- Kicking off the New Year with remarkable achievements, Chengdu-based WestGene made a significant impact at the 3rd mRNA-Based Therapeutics Summit in Berlin, Germany.

Key Points: 
  • CHENGDU, China, Jan. 24, 2024 /PRNewswire/ -- Kicking off the New Year with remarkable achievements, Chengdu-based WestGene made a significant impact at the 3rd mRNA-Based Therapeutics Summit in Berlin, Germany.
  • Renowned for her pivotal role in mRNA research, Dr. Xiangrong Song, co-founder and CEO of WestGene, presented the latest advances in the company's oncology sector, with a special focus on mRNA-based cancer vaccines.
  • The Summit, held January 23-25, is a key event in the field of mRNA drug development, attracting over 200 specialists from various parts of the world.
  • Founded by two visionary scientists, Dr. Wei Yuquan and Dr. Xiangrong Song, WestGene Biotech is dedicated to pioneering the field of mRNA therapeutics.