Increased CGT demand could fuel demand for alternative cleanroom arrangements

[Image courtesy of Azzur]

The continued growth of the cell and gene therapy (CGT) market will increase demand for alternative cleanroom arrangements, predicts Ravi Samavedam, chief innovation officer at Azzur Group (Hatboro, Pennsylvania). “Keeping up with market forces will require an increase in demand for therapies that will outstrip current means of production,” Samavedam said. “Building facilities requires validation that can delay the manufacturing process while CDMOs are already operating at capacity. Neither alternative will satisfy growing demand.”

As a case in point, Charles River expanded its cell therapy CDMO facility in Memphis, Tennessee, earlier this month. Suited for clinical and commercial cell therapy manufacturing applications, the new space has nine new processing suites, adding to an existing 16 cleanrooms. 

Growing demand for CGT could drive similar growth across the sector. Worth $7…

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AWS launches Amazon Omics to sift through multiomic data

At AWS Re:Invent in Las Vegas, Amazon (Nasdaq:AMZN) subsidiary AWS launched Amazon Omics to help researchers sift through genomic, transcriptomic and proteomic data.

The volume of such data is exploding. The National Human Genome Research Institute estimates that researchers will need approximately 40 exabytes to store genome-sequence data generated internationally by 2025. One exabyte equates to one billion gigabytes. A single human genome alone has around three billion base pairs of DNA.

The Amazon Omics platform is now available in North Virginia, Oregon, Ireland, London, Frankfurt and Singapore.

Amazon’s rival Microsoft (Nasdaq: MSFT) has similar offerings known as Microsoft Genomics and Microsoft Immunomics on its Azure cloud.

AWS notes that with Amazon Omics, it is possible to import and standardize petabytes of data to facilitate analytics with a few clicks of the Amazon Omics console.

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Genomenon collaborates with rare disease foundations on drug development

The AI-driven genomics company Genomenon has announced that it will work with COMBINEDBrain, SynGAP Research Fund and SLC-6A1 Connect.

COMBINEDBrain is a 501(c)(3) company focused on developing new treatments for neurodevelopmental disorders. COMBINEDBrain partners with more than 30 neurodevelopmental rare disease foundations, including SynGAP Research Fund and SLC6A1 Connect. “Our collaboration with Genomenon represents a big step toward finding a cure for genetic developmental disorders,” said COMBINEDBrain founder Dr. Terry Jo Bichell in a news release. “We are excited for what the future holds.”

The SynGAP Research Fund is a 501(c)(3) public charity focused on improving the quality of life of people with SYNGAP1-related non-syndromic intellectual disability, a rare genetic disorder associated with an SYNGAP1 gene variant. “Kids with SYNGAP1 are disabled for life and require huge investments and sacrifices from their families,” said Mike Gragli, SYNGAP1 foun…

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How can we target emerging oligonucleotide therapies for success?

[Oleksandr Koniev, CC BY-SA 4.0 via Wikimedia Commons]

The potential of oligonucleotide therapeutics is vast, as illustrated by the growing number of drugs coming to market and the increase in noise and interest in this field. Oligonucleotide therapeutics hold promise to silence aberrant expression, modulate gene splicing or activate the expression of previously undruggable targets across a range of diseases. As the field has progressed, increased understanding has allowed researchers to overcome significant challenges in therapeutic function, from half-life control and extension to target specificity. Despite these advances, the primary challenge remains of targeted delivery to a specific cell type or tissue.

Encapsulation to direct conjugation: A new dawn in targeted delivery

Many have focused delivery around viral vectors and lipid nanoparticles (LNP) to encapsulate the oligonucleotide and protect it from deg…

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Hemophilia gene therapy Hemgenix sets record for world’s most expensive drug 

FDA has approved CSL Behring’s (ASX: CSL) Hemgenix (etranacogene dezaparvovec), an adenovirus-associated virus–based gene therapy for adults with hemophilia B.

The price tag for the gene therapy — the first for hemophilia B — is $3.5 million. The drug is administered as a single infusion.

Hemophilia B is a genetic bleeding disorder caused by missing or defective blood coagulation factor IX, a clotting protein. The condition affects men more often than women.

People with moderate to severe hemophilia B typically receive regular prophylactic infusions of factor IX replacement therapy, frequently several times per week.

In the Phase 3 HOPE-B study, Hemgenix demonstrated “a profound and impressive decrease in bleeding rates compared to standard of care,” said Dr. Steve Pascoe, CSL Behring’s senior vice president of clinical and therapeutic strategy, in an R&D investor briefing.

“We also saw that factor IX level…

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Combatting the rise of antimicrobial resistance 

Methicillin-resistant staphylococcus aureus (MRSA) bacteria. [Image courtesy of NIH]

Antimicrobial resistance (AMR) is a growing global threat that, without intervention, could lead to as many as 10 million deaths annually by 2050.1 Unfortunately, the development of new antibiotics that could combat this risk is a significantly underinvested area, with few new treatments in the pipeline, and other therapy areas currently offering more attractive opportunities for investors. The Basel area in Switzerland is emerging as a leading region for pioneering research into new antimicrobial therapies, thanks to a spirit of innovation, cooperation and collaboration between investors, pharma, biotech, and health organizations. This article highlights the novel approaches to drug-resistant infections emerging from Basel. It discusses how stakeholders across the industry must play their part in finding a solution to the worldwide ch…
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Why the TAAR1 agonist ulotaront holds promise for schizophrenia

Ulotaront (SEP-363856), a trace-amine associated receptor 1 (TAAR1) agonist in Phase 3 clinical development, could prove to be a breakthrough therapy in treating schizophrenia.

In 2020, the New England Journal of Medicine (NEJM) published results from a four-week pivotal study focused on ulotaront, which Sunovion discovered in collaboration with Psychogenics. Ulotaront is the first schizophrenia drug candidate to target TAAR1 rather than the dopamine receptor D2 that has entered randomized controlled trials. The drug candidate also has agonist activity on serotonin 1A receptors.

FDA granted the drug candidate Breakthrough Therapy Designation for the treatment of schizophrenia in 2019.

A new class of medicines

Ulotaront molecule [Image courtesy of PubChem]

“We think [ulotorant] is the first in an entirely new class of medicines,” said Ken Koblan, chief scientific officer of Marlborough, Massachusett…
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Why GSK is pulling multiple myeloma drug Blenrep from U.S. market

FDA has requested that GSK plc (LSE/NYSE:GSK) withdraw the relapsed and refractory multiple myeloma drug Blenrep (belantamab mandolin-blmf) from the U.S. market.

GSK announced in a statement that it has initiated the process.

On November 7, the company announced that the drug did not meet its primary endpoint of progression-free survival (PFS) in the Phase 3 DREAMM-3 study. The humanized IgG1κ monoclonal antibody thus did not comply with FDA’s accelerated approval requirements.

In the study, Blenrep was pitted against Bristol Myers Squibb’s (Nasdaq:GILD), Pomalyst (pomalidomide) and dexamethasone.

GSK shares were up about a percentage point to $33.78.

The FDA approval covered patients with relapsed or refractory multiple myeloma treated with at least four earlier therapies, including an anti-CD38 monoclonal antibody, a proteasome inhibitor, and an immunomodulator.

FDA notes that Blenrep is a first-in-class treatment.

GSK will …

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Fujifilm spending $188M on new cell culture media plant

Fujifilm announced today that it invested $188 million to establish a cell culture media manufacturing facility in Research Triangle Park, North Carolina.

Tokyo-based Fujifilm said it plans for its Fujifilm Irvine Scientific subsidiary to operate the new site. The subsidiary offers the development and manufacturing of advanced cell culture solutions for life science research, bioproduction, cell therapy manufacturing, and medical applications.

According to a news release, the state-of-the-art, 250,000-square-foot facility stretches over 64 acres. It supports the manufacturing of animal component-free, dry powder and liquid media. This adds production capacity of 800,000 Kg/year for dry powder, 3,300,000 L/year for liquid, and 40,000 L/day of water for injection.

“Fujifilm’s intensive investment in the life sciences field represents our commitment to be the dedicated partner for customers in the biopharmaceutical industry,” said Teiichi Goto, president an…

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Eisai prepares to share more findings for Alzheimer’s antibody lecanemab

Tokyo-headquartered Eisai Co. is preparing to share efficacy, safety and biomarker findings from its Phase 3 confirmatory clinical study of the amyloid beta-protein inhibitor lecanemab.

The company will present full results from the Phase 3 confirmatory Clarity AD study of lecanemab in patients with early Alzheimer’s disease in a session at the Clinical Trials on Alzheimer’s Disease conference on November 29.

Eisai is jointly developing the antibody with Biogen (Nasdaq:BIIB).

Last week, Roche (SWX:ROG) that its Alzheimer’s hopeful gantenerumab flunked two Phase 3 studies, failing to meet the primary endpoints in either one.

Gantenerumab won breakthrough therapy designation from FDA in 2021.

In late September, Eisai and Biogen reported positive Phase 3 trial results for lecanemab, noting that the drug candidate met primary and key secondary endpoints. Lecanemab recipients had a 27% reduction in disease progression with a p-…

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Q&A: Getting solid form selection right in early drug development

[Image courtesy of Artem Egorov via iStock Photo]

A significant milestone in early pharmaceutical development is the selection of the lead solid form. This step can be tricky, however, because about 90% of organic molecules have multiple forms. But problems at this stage can spell trouble down the line for drug candidates’ manufacturability, stability and bioavailability.

Because many smaller drug developments don’t have the physical assets needed to screen for solid forms, it is common to work with external partners at this stage.

To learn more about screening for solid forms, we interviewed Michael Morgen, R&D director of Bend, Oregon–based Lonza Bend Small Molecules, a subsidiary of Lonza Group AG (SWX:LONN).

In the following interview, Morgen discusses the solid form landscape and advises selecting the most suitable chemical and physical form of an API. Morgen also explains how …

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Senators Booker and Paul aim to reschedule psilocybin and MDMA to foster research

Cory Booker and Rand Paul [Images courtesy of Senate.gov]

U.S. Senators Cory Booker (D-N.J.) and Rand Paul (R-KY) have drafted the Breakthrough Therapies Act, which would enable the Drug Enforcement Agency (DEA) to research rescheduling Schedule I drugs such as psilocybin and MDMA to Schedule II.

Rescheduling would make it easier for pharmaceutical companies to investigate the therapeutic potential of psilocybin and MDMA.

An aim to facilitate research

“Unfortunately, regulatory red tape and a series of bureaucratic hurdles involved in studying Schedule I substances impedes critical research on these and other promising Schedule I compounds,” said Senator Booker in a news release. “This bill reduces these unreasonably burdensome rules and regulations that delay or prevent researchers from studying – and patients from accessing – this entire class of potential medicines.”

“This bill will make it easi…

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