Lilly ink deal with MiNA; Appia Bio aims for CAR-T with $52M raise; Nuvalent raises $135M; Orchard gene therapy benefits kids

Posted on May 13, 2021 by Delveinsight

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Lilly, MiNA ink agreement to upregulate proteins using RNA

Eli Lilly will utilise the power of MiNA Therapeutics’ proprietary small activating RNA (saRNA) technology platform to develop five novel drug candidates against diseases across the company’s core areas of focus.

Eli Lilly declared a drug development collaboration that could be worth up to USD 245 million per target for the London-based MiNA Therapeutics Limited. 

Under terms of the deal, MiNA will use its saRNA platform to research up to five targets chosen by Lilly. The Indianapolis-based company will then be responsible for the preclinical and clinical development of candidates and will retain exclusive commercialisation rights for any products resulting from the collaboration. 

Eli Lilly will make a payment to MiNA for USD 25 million in upfront cash. The company will then be eligible for an additional USD 1.225 billion and potential royalties on any products from the deal that become commercialised. Eli Lilly did not declare the disease indications it planned to go after with this agreement in its announcement. 

Small activating RNAs are small oligonucleotides designed to work at the gene level and restore a cell’s biology. saRNAs can upregulate intracellular or secreted proteins for therapeutic benefit and have been shown to raise protein levels for both naturally expressed and epigenetically silenced targets. Over the past decade, research has been demonstrated that saRNAs can activate a wide variety of genes in mammals.

Appia Bio raises USD 52 Million series A round to lead the way on CAR-iNKT cell therapy

Appia Bio launches with USD 52 million to develop off-the-shelf cell therapies for cancer-based, hematopoietic, or blood-forming stem cells. Its platform, dubbed ACUA, permits the programming of stem cells to become invariant natural killer T (iNKT) cells, a powerful subtype of T cell. 

Though everyone has iNKT cells in their body, these cells are exceedingly rare. They make up just a fraction of white blood cells found in the peripheral blood, so it is difficult to produce them traditionally, drawing blood and expanding those cells; JeenJoo Kang, PhD, CEO of Appia Bio said.

She further said that [iNKT] cells have multiple tumour cell-killing mechanisms through the natural killer and T cell pathway. They will engineer them with a CAR, and they ponder over being positive because it can address more heterogeneous tumour antigen presentations and get around antigen loss. 

Antigen loss or antigen escape is a process in which cancer cells cease expressing the antigen; the CAR-T is designed for hunting down, such as CD19. Targeting more than one antigen could ruin that escape mechanism and prevent relapse. It could also enhance the odds of cell therapy working in solid tumors, a significant pain point.

Besides solving these problems, Appia also plans to address the barrier of access. Autologous cell therapies are complex, expensive and time-consuming to make. Some patients do not have enough T cells or T cells of good enough quality to make those treatments.  

Producing large amounts of iNKT cells from stem cells that can be stored until they are needed could make cell therapy available to more patients and at a lower cost.

Nuvalent Raises USD 135 Million in Series B Funding

Nuvalent has raised USD 135 million to take ROS1 and ALK kinase inhibitors into the clinic. The series B, which comes months after Nuvalent unveiled a USD 50 million round, sets Nuvalent up to understand drugs designed to improve products such as Roche’s Alecensa and Pfizer’s Xalkori.

Kinase inhibitors have enhanced cancer outcomes by binding to enzymes that drive the proliferation, survival, and metastasis of tumors. However, changes to the binding sites lead to resistance, creating requirements for effective treatments in patients who end responding to current drugs. Nuvalent is working to proffer those treatments.

The work has gained the eyeballs of a clutch of well-known investors. Bain Capital Life Sciences led the series B round with the help of fellow new investors, including Fidelity Management and Research. Deerfield Management, the sole investor in Nuvalent’s series A, also participated.

Nuvalent will utilise the money to fund the clinical development of its parallel lead programs, NVL-520 and NVL-655. The Massachusetts-based biotech is moving the brain-penetrant candidates into the clinic on the strength of preclinical evidence that they are free from some of the limitations of existing rival products.

Gene therapy a potential cure for children born without an immune system

Researchers have created a gene therapy that successfully treated 48 out of 50 children with a form of severe combined immunodeficiency, which leaves them without an immune system.

The study led by an international team of researchers at Great Ormond Street Hospital (GOSH) and the University of California, Los Angeles (UCLA), was posted in the New England Journal of Medicine.

Severe combined immunodeficiency due to adenosine deaminase deficiency, also known as ADA-SCID, is a rare, life-threatening disease that stops children from living everyday life. It is due to the mutations in the gene, which creates the enzyme adenosine deaminase that is vital to a functioning immune system.

Children with ADA-SCID have no immune system and, if left untreated, the condition can be deadly within the first two years of life. Lately, newborn screening for SCID has been implemented in some countries to diagnose the disease early in life.

The standard ADA-SCID treatment comprises once or twice weekly injections of the ADA enzyme until a matched bone marrow donor can be available. If a matched bone marrow donor is not found, patients need lifelong ADA injections along with preventative medicines. These treatments are expensive and hence, out of reach for patients in many countries.

If authorized, gene therapy would be a new treatment option for ADA-SCID as it is a one-time procedure that can provide life-long results.

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