On September 28, 2020 ROME Therapeutics, a biotechnology company harnessing the power of the repeatome in drug development, reported that it has been named by Fierce Biotech as one of 2020’s Fierce 15 biotechnology companies, designating it as one of the most promising private biotechnology companies in the industry (Press release, Rome Therapeutics, SEP 28, 2020, View Source [SID1234567686]).

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ROME was founded to develop novel therapies for cancer and autoimmune diseases by harnessing the power of the repeatome – vast stretches of uncharted genetic material that have long been dismissed as "junk DNA." ROME was launched in April 2020 with $50M Series A funding led by GV and ARCH Venture Partners with participation from Partners Innovation Fund.

"We are honored to be selected as a Fierce 15 company, recognizing ROME’s mission to unlock the uncharted territory of the repeatome and discover a new class of therapies for patients with serious debilitating diseases," said Rosana Kapeller, M.D., Ph.D., CEO, President and Co-founder of ROME Therapeutics. "In a short amount of time, our growing team of ‘Romans’ has generated significant momentum for our lead programs and research platform. We are thankful for the continued support of our founders, collaborators, advisors and investors in pursuit of this mission."

The Fierce 15 celebrates the spirit of being "fierce" – championing innovation and creativity, even in the face of intense competition. This is Fierce Biotech’s 18th annual Fierce 15 selection.

On September 23, 2020 Sapreme, a biotechnology company focused on improving the delivery and efficacy of macromolecule therapeutics, reported positive preclinical data on its proprietary endosomal escape platform in two presentations at the 16th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), held virtually from September 27th to 30th, 2020 (Press release, Sapreme Technologies, SEP 28, 2020, View Source [SID1234567684]).

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Sapreme is developing macromolecule delivery methods based on compounds that release therapeutic cargo from the endo-lysosome, improving access to intracellular targets and enhancing the therapeutic window for these therapeutics. Current macromolecular biologics rely on receptor-mediated endocytic uptake into the endosome and inefficient passive release from these vesicles into the cell to achieve therapeutic efficacy. The company’s presentations demonstrate that Sapreme’s SPT001 compound improves intracellular release of targeted antisense oligonucleotides (ASOs) and thereby also their therapeutic efficacy. In addition to ASOs, SPT001 has also been demonstrated to enhance delivery of other targeted payloads such as antibody-conjugated toxins.

"The data presented today underscore the broad potential of our platform to overcome endosomal entrapment and improve the therapeutic window of macromolecule therapeutics," stated Guy Hermans, Ph.D., Chief Executive Officer of Sapreme. "We are encouraged to see that conjugating SPT001 to liver or tumor targeted ASOs leads to significantly improved silencing, with positive implications for development of metabolic syndrome and oncology targeting drug developments. These results support the further development of SPT001 as the delivery mechanism of choice for future intracellularly active macromolecular drug candidates."

As described in the presentations, multiple in vitro preclinical studies were conducted demonstrating the broad potential of SPT001. Highlights from the data include:

In one experiment, an ASO targeting HSP27 was tested separately or conjugated to monoclonal antibody (mAb) Cetuximab, an epidermal growth factor receptor (EGFR) targeting antibody. While antibody targeting slightly improved HSP27 silencing, results from the study demonstrated that the additional conjugation of SPT001 to the ASO-mAb compound resulted in a highly improved and target-dependent reduction of HSP27 expression.
Further studies showed that combining Cetuximab-SPT001 with Cetuximab-ASO conjugates similarly improved ASO delivery efficacy – demonstrating SPT001 can also improve delivery of an ASO not directly linked to it. Also, combined treatment by Cetuximab-SPT001 and Trastuzumab-ASO of EGFR/Her2 double positive cells yields similar synergies. This dual targeting approach allows for the introduction of additional tumor cell selectivity. Furthermore, such combined Trastuzumab-ASO delivery was effective in double positive cells expressing only low levels of Her2, which are typically not sensitive to Trastuzumab-ASO in the absence of SPT001.
In a third study, Sapreme evaluated the effect of using SPT001 conjugates to improve oligonucleotide delivery to liver cells. N-Acetylgalactosamine (GalNAc) is used as a targeting ligand to drive liver uptake of many oligonucleotides in many late stage clinical trials. ApoB overexpression is known to result in lipid-induced endoplasmic reticulum stress and insulin resistance in the liver, and efficient silencing of ApoB is therefore of therapeutic interest. High doses of GalNAc-ApoB silencing ASOs were required to reduce ApoB expression, whereas combination treatment with GalNAc conjugated SPT001 allowed for complete gene silencing at considerably reduced doses. Similarly, combining SPT001 and ASO payloads with GalNAc into a single next generation compound, resulted in a molecule of superior potency as compared to the GalNAc-ApoB benchmark. Reduced ApoB protein production levels confirmed mRNA silencing results, demonstrating SPT001 conjugates can improve on existing compounds in combination therapy, or serve as building blocks for next generation drug candidates.
The presentations are available on demand at the 16th Annual Meeting of the OTS conference website through this link.

On September 28, 2020 TILT Biotherapeutics, a clinical-stage biotechnology company developing cancer immunotherapeutics, reported it has dosed two patients in a phase 1 clinical trial of its dual cytokine armed oncolytic adenovirus, TILT-123, in Denmark’s Herlev hospital in Copenhagen (Press release, TILT Biotherapeutics, SEP 28, 2020, View Source [SID1234567683]). Of the two patients dosed, both have now passed the trial’s 36-day primary safety endpoint having received three administrations of TILT-123 at the lowest dose.

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TILT-123’s clinical trial (1) is a phase 1, open-label, dose-escalation study of the company’s oncolytic adenovirus coding for Tumor Necrosis Factor Alpha (TNF alpha) and Interleukin 2. In this ‘TUNINTIL’ trial, up to 15 patients with metastatic melanoma will receive three injections of TILT-123 as an initial monotherapy over one month, followed by multiple administrations of TILT-123 plus up to two administrations of tumor infiltrating lymphocytes.

TILT-123 has been engineered to encode two human immunostimulatory cytokines, Tumor Necrosis Factor (hTNFa) and human interleukin 2, in order to direct a powerful T cell response to selectively destroy cancer cells.

TILT Biotherapeutics’ CEO, Akseli Hemminki, a biotech entrepreneur and cancer clinician who has personally treated almost 300 patients with ten different oncolytic viruses, said, "We are putting our recent financing of EUR 6m to good use by progressing our innovative cancer immunotherapies into the clinic. The heart of our approach revolves around the use of armed oncolytic adenoviruses, using cytokines to boost the patient’s immune response, better enabling it to find and destroy cancer cells. We are delighted to be working with Denmark’s prestigious National Center for Cancer Immune Therapy to initiate our first-in-human phase one trial of TILT-123 and look forward to starting additional sites in Finland and France for the TUNINTIL trial. This should act as a springboard to more European trials, and our first US trials, in other solid tumors."

Principal Investigator Inge Marie Svane, from Denmark’s National Center for Cancer Immune Therapy at Herlev Hospital, Copenhagen University, said, "The Nordics have some of the world’s most efficient & innovative healthcare system, and we are excited to work with Finland’s TILT Biotherapeutics to trial its next generation viruses to boost the body’s ability to fight a range of cancers. Our TUNINTIL trial is for metastatic melanoma, which is difficult to treat, and our hope is the trial will help expand the range of therapeutic options available to clinicians and patients, in this and other cancers."

The TUNINTIL trial’s primary objective is to evaluate the safety of TILT-123 and is designed to also deliver insights about the behavior of TILT-123 in humans, such as systemic tumor transduction and virus replication in the tumor, as well as immunological responses. Overall, the trial has the potential to increase the efficacy of adoptive T-cell therapy, remove the need for toxic pre- and post-conditioning regimens, and deliver the combined anti-tumor benefits of armed oncolytic viruses and T-cell therapy. The trial will examine both intra-venous and intra-tumoral delivery of TILT-123 and is due to complete in 2021.

On September 28, 2020 Moderna, Inc., (Nasdaq: MRNA) a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, reported that Dr. Stephen Hoge, President, will participate in the Chardan Virtual 4th Annual Genetic Medicines Conference on October 5, 2020 at 2:00 p.m. ET (Press release, Moderna Therapeutics, SEP 28, 2020, View Source [SID1234567681]).

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A live webcast will be available under "Events and Presentations" in the Investors section of the Moderna website at investors.modernatx.com. A replay of the webcast will be archived on Moderna’s website for 30 days following the presentation.

On September 28, 2020 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, reported that management will present at the following investor conferences (Press release, Sangamo Therapeutics, SEP 28, 2020, View Source [SID1234567680]):

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Jefferies Virtual Gene Therapy / Editing Summit
Thursday, October 1st at 12:00 pm Eastern Time
Chardan Genetic Medicines Conference
Monday, October 5th at 8:00 a.m. Eastern Time
Presentations will be webcast live and may be accessed via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. The presentations will also be available on the Sangamo website after the event.