SMALL CAP IDEA: Why 投資家s should 追加する Hemogenyx 製薬のs to their watchlist
In a 最近の article, I said at least 20 micro-cap 生命科学s 在庫／株s were waiting to pop once 利益／興味 returned to the 部門.
投資家s can now 追加する Hemogenyx 製薬のs?to the watch 名簿（に載せる）／表（にあげる） after it pulled off the 近づく impossible by raising $4.2million at a time when 投資家s have 大部分は gone into hibernation.
This feat of resourcefulness, which will 基金 the 初期の 行う／開催する／段階s of a first-in-human 熟考する／考慮する of the 麻薬 developer's lead 資産, HEMO-CAR-T, only tells part of the story.
The pedigree of the 科学の team 支援 the 成果／努力s of Hemogenyx is impressive.
At the same time, the 利益／興味 in the next 世代 of CAR-T ? the type of therapy that Hemogenyx is developing ? amongst big pharma has seen the likes of AstraZeneca, Johnson & Johnson, Abbvie and Novartis 最高の，を越す the billion-dollar threshold in milestone and 王族 支払い（額）s.
The pedigre e of the 科学の team 支援 the 成果／努力s of Hemogenyx is impressive
Upfront 支払い（額）s, usually a good litmus 実験（する） of 'real' 利益／興味 from these 巨人s of the world of 麻薬s, have been 注目する，もくろむ-watering; in some 事例／患者s up to $100million.
The 推論する／理由? CAR-T 申し込む／申し出s something that the healthcare 産業 rarely comes up with ? a curative 治療, showing the world has truly moved on from the 時代 of me-too, once-a-day pills such as statins.
Anyway, before we delve into the nuts and bolts of the Hemogenyx 投資 事例／患者, it is probably 価値（がある） explaining just what CAR-T therapy is and how it might revolutionise the 治療 of 癌s such as 激烈な／緊急の myeloid leukaemia (AML), which is the particular area of 利益／興味 of this UK biotech.
CAR-T, or chimeric antigen receptor T-独房 therapy, redefines the 戦う／戦い against the 病気 by reprogramming the 団体／死体's own natural defences, called T-独房s, to recognise and kill 癌 独房s.
This 前進するd form of immunotherapy 伴う／関わるs 抽出するing T-独房s from a 患者, genetically 工学 them to 的 癌 独房s, and then 再提出するing these 高めるd 独房s into the 患者's bloodstream.
CAR-T has 論証するd remarkable success in 取り組むing 確かな 血 癌s, 申し込む／申し出ing new avenues for 戦闘ing 以前 untreatable 癌s.
にもかかわらず its 可能性のある, much of the 研究 is now 焦点（を合わせる）d on CAR-T therapy's 重要な 味方する 影響s, while broadening its applicability to a wider array of 癌s.
Hemogenyx will use the newly raised 基金s to take HEMO-CAR-T into the clinic to 扱う／治療する AML later this year.
The open-label 熟考する／考慮する will 実験（する） for any 可能性のある 味方する 影響s from 扱う／治療するing people with 血-borne 病気s and 査定する／（税金などを）課す how the therapy interacts with the 団体／死体.
A 第２位 goal or 'endpoint' will be whether any of the 批判的に ill AML 患者s chosen for the 裁判，公判 答える／応じる to the CAR-T infusion.
Under the strict 指導基準s laid 負かす／撃墜する by the US Food & 麻薬 行政, 患者s will be dosed 個々に one month apart, recognising people are 存在 治めるd with a 潜在的に 有毒な 独房s.
Hemogenyx hopes to have the results from 'two, or even three 患者s' in time for the American Society of Hematology 会議／協議会 in 早期に December.
Success at this 行う／開催する／段階 would be a 30% 返答 率, says Dr Vladislav Sandler, 長，指導者 (n)役員／(a)執行力のある and co-創立者 of Hemogenyx.
Investigational work will be carried out by a team from the University of Pennsylvania, led by Professor Nolle Frey, who has 長，率いるd a number of important CAR-T 臨床の 裁判，公判s.
The entre was 供給するd by Professor Carl June, the 'father' of CAR-T, and his 同僚, Dr Saar Gill, who was 伴う／関わるd in the preclinical 研究 on HEMO-CAR-T.
On the university's 関与, Sandler said: 'This isn't a 状況／情勢 where we've paid [UPenn] to carry out the work. They don't w ork like that. All along it been data driven.'
While no 保証（人） of success, it 供給するs validation for the science behind the 成果／努力s of Hemogenyx.
So expensive is the 過程 of creating individual CAR-T 治療s, that cash raised in February may be enough to dose three 患者s.
Hemogenyx will look to その上の 非，不，無-dilutive 基金ing to 査定する／（税金などを）課す a cohort of around 18 AML 苦しんでいる人s, which will take it to the 段階 II 行う／開催する／段階 of the 臨床の evaluation.
This is 重要な, as it is the point that big pharma has 重さを計るd into the 部門 with the 取引，協定s について言及するd above.
If all of this 示唆するs that Hemogenyx is a one-trick pony, then that would be 誤って導くing.
Before moving into the area of CAR-T, its team together with Eli Lilly, developed a 打開 that 高めるs bone 骨髄 and 茎・取り除く 独房 移植（する） 手続きs.
The approach 焦点（を合わせる）s on the 初期の 批判的な 行う／開催する／段階s of eradicating 病気d 独房s and 取って代わるing them with healthy ones, using a CDX bi-明確な／細部 antibody that is both safer and more 効果的な than 伝統的な chemotherapy.
'We decided to do something that other people wouldn't touch at that time,' says Sandler. This has been a 主題 with all of Hemogenyx's 研究 - 試みる／企てるing the difficult.
As such, the company has created and is a leader in the field of chimeric bait receptors. Put 簡単に, CBRs are designed to attract and catch harmful 独房s, like 癌 独房s, by pretending to be something the harmful 独房s want to attach to.
Once the bad 独房s latch の上に these bait receptors, the 免疫の system can easily find and destroy them.
Its 研究 is 存在 （軍隊を）展開する，配備するd to 戦闘 brain 癌s such as glioblastoma and neurodegenerative 病気s where 配達するing 治療力のあるs across the 血-brain 障壁 has long been problematic.
At the same time, it is working on intranasal 配達／演説／出産 of CBRs to 取り組む 空輸の viral 感染s, an area of 研究 germane to the civil defence 産業 and the 軍の.
While Sandler wouldn't be drawn on 正確に／まさに who he is talking to, there are discussions with parties 利益／興味d in helping develop the viral 感染s 弁護 （軍隊などの）展開，配備, the CEO 確認するd.
'We are talking to 会・原則s and 機関s,' Sandler says. 'There is a lot of 利益／興味 and we are moving these conversations along.'
The Ice Age-like big 凍結する across the small-cap market in the UK means 非，不，無 of company's 可能性のある has been 逮捕（する）d in the 現在の 株 price, which values Hemogenyx at a 取引 地階 £21million.
If we 受託する this is a cyclical anomaly, the 回復する could be 劇の - not just for the 部門, but individual 在庫／株s such as Hemogenyx.
